Let-7 derived from endometrial extracellular vesicles is an important inducer of embryonic diapause in mice – Science Advances

INTRODUCTION

Embryonic diapause refers to a reversible arrest of the development of blastocysts. In wildlife, the phenomenon ensures that the young are born in a favorable environment. Embryonic diapause can be induced in a laboratory condition and is best studied in rodents. In mice, ovariectomy in the early morning of day 4 post-fertilization followed by progesterone administration induces embryonic diapause, during which the blastocysts become dormant and do not implant in the uterus, but can implant and develop normally when reactivated by a single dose of estradiol (1). A recent study showed that sheep blastocysts, which do not normally undergo embryonic diapause, could be induced to become dormant in the uterus of delayed implanting mice and could be reactivated when transferred back to the uterus of the ewe (2), suggesting that embryonic diapause is an evolutionary conserved phenomenon.

The onset, maintenance, and termination of embryonic diapause are under maternal control (3). Various factors regulate embryonic diapause. For instance, photoperiod influences embryonic diapause in minks and lactation affects that in rodents (3). High levels of uterine anandamide maintain embryonic diapause in mice (4), while the metabolite of estrogen, catechol estrogen (5), and polyamines (6) reactivates the dormant embryos in mice and mink, respectively. However, the factors that induce the onset of embryonic diapause in vivo remain unknown.

Let-7 is a family of microRNAs up-regulated in the dormant mouse blastocysts (7). Their expressions return to a low level after estrogen-induced reactivation (8). Although an overexpression of let-7a suppresses implantation (7), the origin and role of let-7 in the dormant blastocysts are unknown. As embryonic diapause is mainly a maternally controlled event and endometrial cells produce extracellular vesicles (EVs) containing microRNAs (9), we tested the hypothesis that endometrial cells produced let-7 containing EVs to induce embryonic diapause in this report. The mechanisms of the action of let-7 on embryonic diapause were investigated. In addition, we provided evidence suggesting that the actions of let-7 on embryos were conserved in humans.

To test the hypothesis, the precursor of let-7a (pre-let-7a) or scrambled RNA (control) was electroporated into mouse blastocysts on day 4 of pregnancy. The level of mature let-7a was >40-fold higher in the pre-let-7a blastocysts than in the control blastocysts (fig. S1A). After 3 days of culture (day 7), the level of let-7a remained threefold higher in the pre-let-7a group (fig. S1A). The majority of the pre-let-7a blastocysts (95 3.2%) were morphologically viable, with a large blastocoel (Fig. 1A), whereas 31 2.2% of the control embryos had shrunken in size and some degenerated by that time. On day 12, over 50% of the embryos with a pre-let-7a overexpression remained morphologically viable, while all the control embryos had degenerated (Fig. 1A).

(A) Overexpression of let-7a extended the survival of blastocysts in culture. Survived blastocysts were defined as those with a well-defined blastocoel. (B) The percentage of apoptotic cells was low in blastocysts with overexpression of let-7a on day 7 (D7-let7) and was comparable to that in day 4 activated (D4-act) blastocysts. Blastocysts electroporated with scrambled RNA (D7-scr) had significantly higher percentage of TUNEL+ cells. *P < 0.05. (C) Glucose metabolism of the D7-let7 and in vivo dormant (Dor) blastocysts was low with significant reduction in glucose, pyruvate uptake, and ATP levels when compared with the D4-act blastocysts and E2-induced reactivated blastocysts (E2-act), respectively. D7-let7 had a high lactate production, which was low in the dormant blastocysts. *P < 0.05. (D) Expression of genes related to glucose metabolism. (E and F) The percentages of proliferating (Ki67+, green) cells (E) and cells with DNA synthesis (EdU+, green) (F) were lower in blastocysts of D7-let7 when compared to that in D4-act blastocysts. Numbers in parenthesis are number of embryos analyzed. *P < 0.05. (G) Dor, D7-let7, and D7-scr blastocysts did not bind significant amount of FITC-labeled EGF. The binding of EGF was high in the D4-act blastocysts and the delayed implanting mice 6 hours after E2-act.

Consistent with the above observation, the percentage of apoptotic [terminal deoxynucleotidyl transferasemediated deoxyuridine triphosphate nick end labelingpositive ( TUNEL+)] cells in the day 7 blastocysts (N = 6 embryos per group) with electroporation of pre-let-7a on day 4 (D7-let7) was significantly lower than that of the control embryos (D7-scr, P < 0.05; Fig. 1B). Blastocysts collected in the afternoon of day 4 of pregnancy (in vivo activated blastocysts, D4-act) had a low percentage of TUNEL+ cells comparable to that of D7-let7 blastocysts (Fig. 1B).

It is well established that metabolism is suppressed in the dormant embryos (10). Therefore, we tested whether an overexpression of let-7a would change the energy metabolism of embryos. As expected, D7-let7 and in vivo dormant (Dor) blastocysts had low glucose metabolism; their glucose, pyruvate uptakes, and adenosine triphosphate (ATP) levels were significantly lower than those of the D4-act and estrogen-induced reactivated (E2-act) blastocysts, respectively (Fig. 1C; N = 5). The reduced glucose metabolism was likely a result of the inhibitory action of let-7 on its target genes related to glucose metabolism (predicted by TargetScan or RNA22), Hk2, Fbp1, Dld, and Dlst (Fig. 1D). Lactate production was unexpectedly significantly higher in the D7-let7 blastocysts than in the D4-act blastocysts (Fig. 1C), which might be due to a high level of Ldha (lactate dehydrogenase A) mRNA in the former (Fig. 1D).

The effects of an overexpression of let-7a on the proliferation and extent of the active DNA synthesis of blastomeres were determined by immunostaining for Ki67 and 5-ethynyl-2-deoxyuridine (EdU) incorporation assay, respectively. The percentage of Ki67+ cells in the D7-let7 blastocysts (N = 12) was significantly lower than that of the D4-act blastocysts (N = 10, P < 0.05; Fig. 1E). The Ki67+ cells in the D7-let7 blastocysts were concentrated in the inner cell mass; this is consistent with the observation that the mouse trophectoderm cells entered dormancy earlier than the inner cell mass cells (11, 12). Consistent with cell proliferation, the percentage of EdU+ cells was about fourfold higher (P < 0.05) in the D4-act blastocysts (N = 5) than in the D7-let7 blastocysts (N = 7) (Fig. 1F). The observations were in line with the report that cell cycle arrest in the delayed implanting blastocysts occurs before the S phase (13).

An increase in epidermal growth factor (EGF) binding is a marker of reactivation of dormant mouse blastocysts (5). Dormant blastocysts did not bind a significant amount of fluorescein isothiocyanate (FITC)labeled EGF (Dor; Fig. 1G). EGF binding was up-regulated in blastocysts from delayed implanting mice at 6 hours after estradiol-induced reactivation (E2-act). However, D7-let7 and D7-scr blastocysts exhibited a low EGF binding. The similarly low EGF binding of D7-let7 and D7-scr blastocysts could be attributed to different causes; the former reflected a diapause state, while the latter reflected a deteriorating state with many apoptotic cells (Fig. 1B).

To determine whether the overexpression of let-7 in embryos had been in a reversible dormant state in vivo, a 2-fluoroestradial-17 (2-Fl-E2)treated mouse model was used. 2-Fl-E2 treatment induces a receptive state of the endometrium (5). It also inhibits the uterine estrogen-2/4-hydroxlyase activity, thereby suppressing the synthesis of 4-hydroxy-E2, a catechol metabolite of estradiol-17 (E2) required for the activation of dormant mouse blastocysts (5). Thus, 2-Fl-E2 injection induces uterine receptivity but fails to reactivate the dormant blastocysts in the delayed implanting mice (5). We transferred D7-let7 blastocysts into the 2-Fl-E2treated pseudo-pregnant uteri (N = 6 mice per group); the number of implantation sites at 28 hours after embryo transfer was significantly lower than that after the transfer of the D4-act blastocysts (P < 0.05; Fig. 2A).

(A) The implantation rate of D7-let7 after embryo transfer into 2-Fl-E2treated mice was significantly lower than that of D4-act. 2-Fl-E2 injection induces uterine receptivity but fails to activate the dormant blastocysts in the delayed implanting mice. Numbers in parenthesis are number of animals receiving the transferred embryos. *P < 0.05. (B) Live births were obtained after transfer of blastocysts with overexpression of let-7a for 3 days (D7-let7) and 4 days (D8-let7), although the live birth rate was lower than that from D4-act blastocysts. Numbers in parenthesis are number of live birth/total number of embryos transferred. (C) Survival curve of blastocysts carrying an inducible let-7g transgene with and without DOX treatment cultured in KSOM + AA medium. Most of the control embryos survived until day 6, whereas 50% of the DOX-treated embryos maintained good morphology even on day 13. (D) Live births were obtained after transfer of let-7g blastocysts treated with DOX treatment for 3 days (D7+DOX) and 5 days (D9+DOX). No live birth was obtained from transfer of day 7 WT blastocysts. Numbers in parenthesis are number of live birth/total number of embryos transferred. (E) Microarray analyses of the mRNA expression of D4-act (D4-act-7g), in vitro (DOX treatment in culture, DOX-7g), and in vivo induced dormant (Dor-7g) let-7g blastocysts. Heatmap of the top 500 differentially expressed genes between the dormant and D4-act blastocysts was shown. (F) Venn diagram showing the number of differentially expressed genes between in vitro (DOX treatment in culture) or in vivo induced dormant blastocysts against D4-act blastocysts. Photo credit: W. M. Liu, The University of Hong Kong.

The reversible nature of let-7induced embryonic diapause was tested by transferring D7-let7 blastocysts into D3 pseudo-pregnant uteri. Live births were obtained from blastocysts with an overexpression of let-7a for 3 days (D7-let7) and 4 days (D8-let7; Fig. 2B). All the pups had normal birth weight (fig. S1B). No pup was born after the transfer of the D12-let7 and D7-scr blastocysts (data not shown).

In the above experiments, electroporation induced only a transient rise in the let-7a level for a few days. To determine the prolonged action of a high level of let-7 on embryo survival in vitro, we produced transgenic mice from embryonic stem cells with doxycycline (DOX)inducible let-7g (a gift from G. Q. Daley, Harvard Stem Cell Institute, Boston, MA, USA). DOX treatment induces let-7g expression in the transgenic mice. The transgene is unique in that the loop region of the precursor let-7g (pre-let-7g) in the transgene is replaced by that of microRNA-21. Therefore, endogenous Lin28 cannot bind to pre-let-7g and block let-7g biogenesis in the transgenic mice (14). Let-7 family members contain a similar seed sequence that spans from nucleotide 2 to 8 in mice (15). This conserved feature suggests that the let-7 family members have similar target mRNAs and functions. Identical changes in the expression of 14 genes were observed in the blastocysts after an overexpression of let-7a and let-7g (fig. S2). The above embryo transfer experiment was repeated with the transgenic mice.

When blastocysts from the transgenic mice were treated with DOX during culture, the let-7g level in the embryos increased 32-fold, and 50% of the blastocysts survived for 14 days in vitro (Fig. 2C). Pups were obtained after the transfer of DOX-treated day 7 (D7+DOX) and day 9 (D9+DOX) blastocysts, but not day 7 wild-type (D7WT) blastocysts, into WT day 3 pseudo-pregnant mice (Fig. 2D). Because the inducible let-7g transgenic mice better simulated the in vivo condition, their blastocysts were mainly used for subsequent experiments unless otherwise stated.

To understand the molecular actions of let-7 on embryonic diapause, the GeneChipTM Mouse Gene 2.0 ST Array was used to determine the transcriptomes of D4-act, in vivo dormant, and let-7induced dormant blastocysts from let-7gtransgenic mice. Unsupervised hierarchy clustering (Fig. 2E) and principal components analysis (fig. S3A) showed that the mRNA profiles (table S1) of the in vitro induced dormant blastocysts (DOX treatment in culture, DOX-7g) and the in vivo dormant blastocysts (Dor-7g) were similar but distinct from those of the D4-act let-7g blastocysts (D4-act-7g). To confirm the data obtained from the mRNA profiles, the total RNAs isolated from five pools of D4-act-7g, Dor-7g, and DOX-7g blastocysts were subjected to direct quantitative polymerase chain reaction (qPCR) analyses for the transcript levels of six genes, namely, Ccne1, Btg1, Pkm, Oxct1, Fbp1, and Sap1 (fig. S3B). These genes were involved in cell cycle (Ccne1 and Btg1), carbohydrate metabolism, energy pathway (Pkm and Oxct1), and chromatin remodeling (Sap30). The expression patterns were consistent with the results of the array. Among them, two genes (Btg1 and Oxct1) were significantly higher, while the rest were significantly lower in the Dor-7g and DOX-7g blastocysts than in the D4-act-7g blastocysts (P < 0.05).

Compared with the D4-act-7g blastocysts, the in vitro and the in vivo induced dormant blastocysts exhibited 3444 and 2452 differentially expressed genes, respectively (Fig. 2F). Among the differentially expressed genes, 1006 of them were common in the two comparisons (Fig. 2F). Gene ontology analysis of these common genes using Database for Annotation, Visualization, and Integrated Discovery (DAVID) showed that they were related to mitotic nuclear division, cell division, G1-S transition of mitotic cell cycle, DNA repair, DNA replication, and cell cycle (table S2).

Because embryonic diapause is a maternally regulated phenomenon, we tested the possibility of a maternal origin of let-7 in the delayed implanting blastocysts. This possibility is supported by three observations. First, transmission and scanning electron microscopy showed EV-like structures in the mouse uterine lumen (fig. S4A) and on the trophectoderm of blastocysts collected in the uterine lumen (fig. S4B), respectively. Second, immunostaining detected the presence of CD63, a marker of EVs, in the uterine epithelium of mice (fig. S4C) and on the surface of EVs from uterine luminal fluid (ULF; fig. S4D). Nanoparticle tracking analysis showed that the EVs in ULF had a mean size of 82.3 nm (fig. S4E). Western blot analysis showed that these ULF-EVs were positive for HSP70, CD63, and TSG101 and negative for calnexin and GM130. Third, mouse ULF contains let-7 carrying CD63+ EVs, which can be internalized by blastocysts (16). To obtain further evidence, the expression patterns of let-7 in the endometrial epithelial cells, EVs from ULF (ULF-EVs), and in blastocysts from mice before embryonic diapause, during embryonic diapause, and after E2-induced reactivation were determined. The results showed that they had similar patterns with a high let-7a expression only in the dormancy period (Fig. 3A). The pattern was markedly different from that of the stromal cells (fig. S5), supporting the idea that the endometrial epithelial cells produced EVs containing let-7a during delayed implantation.

(A) The expression patterns of let-7a in endometrium epithelial cells, EVs of ULF-EVs, and blastocysts in the mouse delayed implantation model before and during diapause and 1, 3, and 24 hours after estradiol-induced reactivation were similar. *P < 0.05; **P < 0.01; ***P < 0.001. (B) The expression of pre-S7gL21 was significantly higher in ULF-EVs from DOX-treated transgenic mice carrying an inducible let-7g transgene relative to that of ICR mice (upper panel) and in ICR blastocysts after transfer into the DOX-treated let-7g transgenic mice when compared to ICR blastocysts without transfer (lower panel). **P < 0.01. (C) Let-7 in EVs suppressed the level of LIN28 in human trophoblast JEG-3 cells. Let-7acontaining EVs generated by transfection of endometrial cells with pre-let-7a, but not EV-free let-7a, suppressed expression of LIN28. *P < 0.05. (D and E) Let-7aenriched EVs suppressed expression of c-myc protein (D) and DNA synthesis (E) in day 4 mouse blastocysts. Day 4 embryos were cocultured with let-7aenriched EVs or control EVs. Expression of c-myc protein (green) was observed by confocal microscopy after immunohistochemical staining and analyzed with the ImageJ software (n = 15 embryos for each group). DNA synthesis was determined by EdU incorporation assay. Green, newly synthesized DNA; blue, DAPI. n = 20 embryos for each group. **P < 0.01. (F) Let-7genriched EVs from DOX-treated endometrial epithelial cells of let-7g transgenic mice (Let-7g-EV) prolonged the survival of WT day 4 blastocysts in KSOM + AA medium. The survival rate on day 7 of untreated blastocysts and those treated with EVs from WT mice was low. Numbers in parenthesis are number of embryos analyzed. *P < 0.05.

The inducible let-7g transgenic mice carry a unique chimeric let-7g Stem/miR-21 loop sequence (S7gL21), in which the loop of pre-let-7g is replaced by that of microRNA-21. qPCR assay was developed to detect the expression of the transgene; the forward primer targeted on a sequence that crossed the stem and the loop of S7gL21 so that only the precursor of the transgene, but not pre-let-7g, was amplified. DOX treatment significantly induced the expression of the sequence in the liver (data not shown) and ULF-EVs (Fig. 3B, upper panel) of the transgenic mice, but not that of the ICR (Institute of Cancer Research) mice. Blastocysts from ICR mice were transferred into the pseudo-pregnant delayed implanting let-7g transgenic mice. Three days later, the expression of the S7gL21 sequence was significantly higher in the transferred embryos than in those that were not transferred (Fig. 3B, lower panel). The result confirmed that let-7 was transferred from the mother to the embryos in vivo.

To study the biological effect of let-7a and let-7g in EVs, we produced let-7enriched EVs by two methods. First, let-7aenriched EVs were isolated from the spent culture medium of human endometrial Ishikawa cells transfected with pre-let-7a. Transfection with scrambled RNA was used as control. The collected EVs at physiological concentration were then used to treat day 4 blastocysts or human trophoblast JEG-3 cells. Endometrial EVs containing let-7a, but not EV-free let-7a, were biologically active in suppressing the expression of let-7 targets, LIN28A [lin-28 homolog A (Caenorhabditis elegans); Fig. 3C], C-MYC (MYC proto-oncogene), and RICTOR (RPTOR independent companion of MTOR, complex 2; fig. S5B) in the JEG-3 cells. Incubation of the let-7aenriched EVs for 24 hours significantly decreased the protein expression of c-myc in the treated blastocysts relative to the control embryos (Fig. 3D) and reduced their DNA synthesis (let-7 EVs; Fig. 3E) to a level comparable to that in the in vivo dormant blastocysts (Dor; Fig. 3E).

To better simulate the in vivo situation, let-7genriched EVs were obtained from endometrial epithelial cells of let-7g transgenic mice treated with DOX for 4 days in a medium supplemented with 10% EV-free fetal bovine serum (FBS) and were used at physiological concentration to treat WT day 4 blastocysts in KSOM + amino acid (AA) medium. After 3 days of culture, 82 16.2% (N = 150) of the let-7g-EVtreated blastocysts still had the blastocoel, and 12 5.4% of them developed to term after an embryo transfer. In contrast, 85 10.1% (N = 100) of the blastocysts degraded in the absence of EVs (Fig. 3F), and the survived ones produced no pups after transfer.

Inhibition of mTOR (mammalian target of rapamycin) (17) or MYC (18) induces a diapause-like state in mouse embryos. We consistently observed significant decreases in the transcript expression of c-myc and Akt1 (mTOR activator) and increases in that of Tsc1 and Tsc2 (mTOR inhibitors) in the let-7ginduced dormant blastocysts when compared with the untreated blastocysts (Fig. 4A). On the other hand, Pten of PIK3 (phosphatidylinositol-3-kinase) signaling, an upstream pathway of mTOR, was unaffected by the DOX treatment.

(A) RT-PCR analysis showing the expression of c-myc and mTOR signaling components after let-7g overexpression. D4 blastocysts from let-7g transgenic mice were cultured in the presence or absence of DOX for 48 hours before determination of gene expression. *P < 0.05. (B) Expression of c-myc in JEG-3 trophoblast cells at 24 hours after transfection of c-myc mRNA. **P < 0.01. (C) Representative confocal images of EdU incorporation (green) in blastocysts at 24 hours after electroporation of c-myc mRNAs or scrambled RNA. Blue, nuclei. Scale bar, 50 m. (D) Expression of c-Myc and pRpS6 in JEG-3 cells treated with mTOR inhibitor, INK-128, for 24 hours. ***P < 0.001. (E) Western blot analysis of the effect of c-myc inhibitor 10058-F4 (MI) on mTORC1 targets (pRpS6 and p4EBP1) and mTORC2 target (pAKT) in JEG-3 cells. All quantification measurements are normalized to -actin. **P < 0.01. (F) Representative confocal images of blastocysts immunostained for p4EBP1 and pRpS6. D4 blastocysts from let-7g transgenic mice were electroporated with or without c-myc mRNA before culture in the presence or absence of DOX. ICR D4 blastocysts (D4) and dormant embryos (Dor) served as positive and negative controls, respectively. (G) Expression of Rictor and phosphorylated AKT protein in JEG-3 cells transfected with either let-7a mimics or scramble for 24 hours. Data are presented as means SE. *P < 0.05; **P < 0.01. (H) Representative confocal microscope images showing Rictor in embryos during diapause and at 6-hour postestrogen-induced reactivation (A6h). Scale bar, 50 m.

Next, we determined whether c-myc mediated the action of let-7g on the induction of embryonic diapause. In vitro transcription was used to generate c-myc mRNA. The mRNA was biologically active, and the level of C-MYC protein in the JEG-3 cells increased fourfold at 24 hours after transfection of the mRNA (Fig. 4B). Electroporation of the c-myc mRNA enhanced C-MYC expression (fig. S6A) and EdU incorporation (Fig. 4C) in D4 blastocysts. Overexpression of c-myc also nullified the effects of let-7g overexpression on the prolongation of the embryo survival in vitro; in the presence of DOX, the let-7g transgenic blastocysts transfected with c-myc mRNA could only survive until day 6; the vast majority died the next day, whereas 50% of the DOX-treated transgenic blastocysts without the transfection survived up to day 15 (fig. S6B).

C-MYC is upstream of mTORC1 in rat fibroblasts (19). The incubation of JEG-3 cells with an inhibitor of mTORC1/mTORC2 pathways, INK-128, for 24 hours reduced phosphorylation of mTORC1 target and RpS6 phosphorylated at Ser235/236 (pRpS6) and had no effect on the expression of C-MYC (Fig. 4D). On the other hand, treatment with the C-MYC inhibitor 10058-F4 [MYC inhibitor (MI)] (20) significantly decreased the phosphorylation of mTORC1 targets, pRpS6, and p4EBP1 (4EBP1 phosphorylated at Thr37/46) in the JEG-3 cells (Fig. 4E) and D4 blastocysts (fig. S6C). The phosphorylation level of mTORC2 target, AKT at Ser473 (pAKT), was unexpectedly not affected (Fig. 4E and fig. S6C), suggesting that mTORC1, but not mTORC2, signaling was downstream of C-MYC in blastocysts and trophoblast cells. Overexpression of c-myc mRNA in day 4 let-7g blastocysts for 48 hours reduced the inhibitory effects of DOX-induced let-7g on mTORC1 targets (Fig. 4F).

Inhibition of mTORC1 alone was insufficient to induce embryonic diapause (17). Therefore, bioinformatics analysis was conducted to find whether let-7 also targeted the mTORC2 pathway. TargetScan identified that a component of mTORC2, Rictor, was a potential target of let-7. The prediction was supported by the down-regulation of RICTOR expression in the JEG-3 cells at 48 hours after transfection of the cells with pre-let-7a (Fig. 4G). Dual luciferase assay confirmed that Rictor was a direct target of let-7a. At 24 hours after transfection, let-7a mimics reduced the luciferase activity of Rictor 3 untranslated region (3UTR) reporter by about fivefold when compared to the scramble control (fig. S7). Mutation of the let-7a binding sites on the reporter construct abolished the reduction in luciferase activity (fig. S7). As expected, the expression of RICTOR was lower in the dormant embryos (Dor) than in those at 6-hour postE2-induced reactivation (A6h; Fig. 4H). The action of let-7 on mTORC2 signaling was further confirmed by a significant decrease in pAKT expression in the JEG-3 cells at 48 hours after transfection of pre-let-7a when compared with the scramble control (Fig. 4G).

The inhibition of polyamine biosynthesis delays the reactivation of dormant embryos in vitro (21). We consistently found an increased expression of polyamine biosynthesis enzymes, ornithine decarboxylase (ODC1) and spermine synthesis enzyme (SMS) in the reactivated embryos (A24h), relative to the dormant embryos (Dor; Fig. 5A).

(A) Representative confocal microscope images of Dor and Act-24h embryos immunostained for ODC1 and SMS. Scale bar, 50 m. (B) Schematic representation of the five putative myc response elements in the proximal Odc1 promoter (upper). Luciferase activities in 293T cells after cotransfection of a c-myc expression vector with empty luciferase reporter (pGL3) and long or short Odc1 promoter constructs (lower). Data represent means SE. ***P < 0.001 versus empty reporter (pGL3); ###P < 0.001 versus long Odc1 promoter construct. (C) ChIP-qPCR assay to evaluate the relative myc binding to the Odc1 promoter isolated from uterine epithelial cells on day 4 of pregnancy (D4-ME) and during diapause (Dor-ME). Amplified putative myc response elements (1, 2, 3, and 4-5) are depicted in the upper panel of (B). Data represent means SE. *P < 0.05 and **P < 0.01. (D and E) The expression of ODC1 was detected at 24 hours after incubation with or without the c-myc inhibitor 10058-F4 (MI) in JEG-3 trophoblast cells (D) and in blastocysts (E). All quantification measurements in Western blot were normalized to -actin. Scale bar, 50 m. (F) D4 blastocysts from let-7g transgenic mice were electroporated with or without c-myc mRNA before culture in the presence of DOX. D4 and Dor blastocysts served as positive and negative controls, respectively. (G) Schematic diagram summarizing the molecular actions of endometrium-derived let-7 in inducing embryonic diapause. Blastocysts take up endometrial EVs enriched with let-7. Let-7 inhibits c-myc, leading to reduction of mTORC1 activity and reduction in ODC1 biosynthesis. Polyamines are required for reactivation of diapausing embryo. Let-7 also suppresses mTORC2 directly by its action on mTORC2 component Rictor.

Odc1 encodes a rate-limiting enzyme in polyamine biosynthesis. An analysis of a genome-wide embryonic C-MYC chromatin immunoprecipitation (ChIP) sequencing dataset (22) revealed recruitment of C-MYC at the promoter of Odc1 and identified five putative c-myc response elements on the promoter (Fig. 5B). Luciferase reporter assays showed that response elements 3, 4, and 5 were the major sites that conferred C-MYC responsiveness (Fig. 5B). Site-specific ChIP assays using the DNA from mouse primary uterine epithelial cells isolated from delayed implanting (Dor-ME) and day 4 activated mouse uteri (D4-act) confirmed the recruitment of C-MYC to these response elements (Fig. 5C). There were reductions in the recruitment of C-MYC to these elements of Odc1 promoter in the uterine epithelial cells during embryonic diapause (Dor-ME) when compared to those from D4-act mice (Fig. 5C).

Our data further showed that the inhibition of C-MYC by MI for 24 hours significantly decreased the protein level of ODC1 in the JEG-3 cells (Fig. 5D) and D4 blastocysts (Fig. 5E). Treatment with DOX reduced the protein expression of ODC1 in the let-7g blastocysts relative to the D4 blastocysts (Fig. 5F). The action was mediated by C-MYC as the transfection of c-myc mRNA abolished the let-7induced down-regulation of ODC1 in the embryos (Fig. 5F). Together, endometrial epithelial cellderived let-7 suppressed c-myc/mTORC1, mTORC2 signaling, and polyamine biosynthesis to induce embryonic diapause (Fig. 5G).

Non-diapause sheep blastocysts become dormant in the uteri of delayed implanting mice and can be reactivated after a transfer to the uterus of ewe (2). We tested whether let-7enriched EVs would affect the differentiation of a human embryo surrogate model termed BAP-EB (23). BAP-EB was derived by BAP (BMP4, A83-01, and PD173074)induced differentiation of embryoid bodies of human embryonic stem cells (hESCs) into trophoblast spheroids. BAP-EB spheroids resemble human blastocysts in size and morphology. They express markers of trophectoderm and trophoblast and do not express those of other germ layers. BAP-EB selectively attached onto primary receptive endometrial epithelial cells and receptive endometrial epithelial cell lines, but not other nonendometrial cell lines, nonreceptive endometrial cell line, and primary prereceptive endometrial epithelial cells (23).

The time when BAP was added to induce trophoblast differentiation was considered as time zero of post-induction of differentiation (pid). During differentiation, the expression of the marker of inner cell mass (OCT4) in the BAP-EB spheroids decreased rapidly at 48-hour pid and was undetectable by 96-hour pid. The expression pattern was different from that of the trophectoderm and trophoblast markers. The trophectoderm marker (CDX2) showed a transient increase at 48-hour pid, while those of trophoblast (CK7, CDH1, and GATA2), syncytiotrophoblast (ERVW-1 and CGB), and extravillous trophoblast (MMP2 and HLA-G) increased progressively with differentiation. Treatment with let-7g-EVs at 48-hour pid significantly affected the mRNA expression of these trophoblast markers at 96-hour pid relative to the control-EVtreated spheroids (Fig. 6A); let-7treated BAP-EB at 96-hour pid exhibited significantly higher levels of trophectoderm marker (CDX2) and lower levels of trophoblastic markers (GATA2, CK7, CDH1, ERVW-1, CGB, MMP2, and HLA-G). The comparable expression levels of these markers in the let-7g-EVtreated spheroids at 96-hour pid with that of the spheroids at 48-hour pid were consistent with a high level of let-7induced dormancy and cessation of differentiation.

(A) Treatment of hESC-derived trophoblast spheroids (BAP-EB, human embryo surrogates) with Let-7genriched EVs at 48-hour pid significantly reduced the mRNA expression of trophoblast [CK7 (keratin 7), CDH1 (cadherin 1), and GATA2 (GATA-binding protein 2)], syncytiotrophoblast [ERVW-1 (endogenous retrovirus group W member 1, envelope) and CGB (chorionic gonadotropin subunit beta 3)], and extravillous trophoblast [MMP2 (matrix metallopeptidase 2) and HLA-G (major histocompatibility complex, class I, G)] markers at 96-hour pid relative to the control EVtreated BAP-EB. The expression of pluripotent genes [OCT4 (POU class 5 homeobox 1) and CDX2 (caudal type homeobox 2)] decreased within this period. *P < 0.05. (B) Treatment with let-7genriched EVs significantly reduced the attachment of BAP-EB onto receptive endometrial epithelial cells, Ishikawa. At 48-hour pid of BAP-EB, control EVs or Let-7genriched EVs were incubated with BAP-EB until 72-hour pid; the spheroids were evenly transferred onto a confluent monolayer of Ishikawa cells and further cocultured for 3 hours. Nonadherent spheroids were removed, and the percentage of attached BAP-EB was calculated. *P < 0.05. (C) Viability of day 5 human blastocysts after treatment with let-7enriched EVs or control EVs for 3 days. Blastocysts with a blastocoel were considered viable.

BAP-EB spheroids attach specifically onto receptive endometrial cells resembling the early implantation event (23). Treatment with let-7g-EVs reduced the protein expression of c-myc (fig. S8) in the BAP-EB and significantly decreased their attachment onto the receptive endometrial epithelial Ishikawa cells (P < 0.05; Fig. 6B).

Good quality human blastocysts can be obtained in 65% of the cultured blastocysts on day 5 and 30% on day 6, but only 5% on day 7 (24). In this study, day 5 human blastocysts (N = 21) were treated with let-7genriched EVs and their viability in terms of the presence of a blastocoel, morphology of the trophectoderm cells, and the inner cell mass cells were examined. The blastocoel and good morphology were maintained in 52% of let-7g-EVtreated blastocysts on day 7, whereas only 30% did so in the untreated group (N = 10). One of the EV-treated blastocysts remained viable until day 8 (Fig. 6C). The observations were consistent with a beneficial effect of let-7 on embryo survival in vitro.

There have long been efforts searching for the natural initiator of embryonic diapause but without much success. Our data show that let-7 of endometrial epithelial origin is a key inducer of embryonic diapause in vivo. Specifically, when the mice undergo diapause, the endometrium generates let-7enriched EVs, which are taken up by blastocysts. Two observations support the role of let-7 in the induction of embryonic diapause. First, the overexpression of let-7a and incubation with let-7enriched EVs prolonged blastocyst survival in vitro. Second, the treated embryos developed to term after an embryo transfer. The study further demonstrates a conserved action of let-7 on the induction of diapause-like phenotype in a human embryo surrogate and the prolongation of survival of human embryos in vitro.

Let-7 is a key inducer of embryonic diapause because it can simultaneously regulate the two known pathways leading to embryonic diapause. Two recent studies showed that simultaneous inhibition of C-MYC and N-MYC (18) or inhibition of mTORC1 and mTORC2 (17) signaling is required for the induction of embryonic diapause. The present data showed that let-7 induced embryonic diapause via the inhibition of both the C-MYC/mTORC1 and mTORC2 signaling pathways. Although the action of let-7 on n-myc has not been studied, it is known that there are two let-7 binding sites in the 3UTR of Mycn (25). Therefore, it is likely that let-7 also suppresses the expression of n-myc.

Let-7 inhibits Odc1 expression via the suppression of c-myc. This can be a mechanism reducing the potential of diapausing blastocysts to be reactivated, as polyamine biosynthesis is required for reactivation, and inhibition of their biosynthesis in embryos delays reactivation in vitro (21). Our site-specific ChIP assay demonstrated a reduced recruitment of C-MYC to the Odc1 promoter during diapause. Whether the reduction is due to the suppression of C-MYC expression resulting from a high expression of let-7 in the cells during diapause remains to be determined.

We localized the C-MYC protein mainly to the cytoplasm of embryos. This differs from the expected nuclear localization of C-MYC in many cell types. The antibody used in this report localized the expression of C-MYC to the nuclei of embryonic stem cells (fig. S6D). Three other antiC-MYC antibodies were tested and showed similar cytoplasmic C-MYC expression in embryos (data not shown). The expression of cytoplasmic MYC has been reported. MYC-nick is a cytoplasmic cleavage product of the full-length C-MYC, widely expressed in a large number of cell lines (26). It is expressed in differentiating cells and tissues and plays a significant role in the differentiation of myofibroblasts and the trans-differentiation of fibroblasts into muscle cells (26). Thus, a let-7induced decrease of cytoplasmic C-MYC may lead to a decrease in the differentiation potential of the diapausing blastocysts.

Let-7 overexpression induced dormancy via the suppression of apoptosis, cell proliferation, DNA synthesis, and energy metabolism. The majority of these phenotypes can be explained by the actions of let-7 on its targets, for instance, a high level of let-7 targets on c-myc to inhibit proliferation (27), caspase-3 to suppress apoptosis (28), and HK2 to reduce glucose utilization (29). A high let-7 expression is also associated with the quiescence of fibroblasts (30). Our results on Hk2 and Fbp1 mRNA expression are in contrast with those of a previous proteomic study (31). The discrepancies could possibly be due to different comparisons made in the two studies: dormant versus reactivated blastocysts at 12 to 14 hours after E2 injection in the previous studies but dormant versus D4 activated blastocysts in the present study.

Contrary to the low lactate production of dormant embryos in vivo, an overexpression of let-7 up-regulated lactate production. Dld and Dlst are let-7 targets. They are subunits of the 2-oxo-glutarate complex and the -ketoglutarate dehydrogenase complex of carbohydrate metabolism. Inhibition of DLD (Dihydrolipoamide dehydrogenase) activity in spermatozoa causes lactate accumulation (32), and infantile lactic acidosis is associated with severe deficiencies of the -ketoglutarate dehydrogenase complex (33). These conditions may have been recapitulated in the present model by the suppressive action of let-7 on Dld and Dlst. Alternatively, the observation could be due to an indirect action of let-7 on Ldha via its well-known target Lin28a. Overexpression of Lin28a decreases the expression of Ldha in the human embryonic kidney cells (34). The molecular mechanism of Lin28a on the observation remains to be determined.

Transcriptomic analyses showed that the mRNA profile of let-7ginduced embryonic diapause is about 30 to 40% similar to that of in vivo dormant embryos. These common genes represent the let-7affected genes contributing to in vivoinduced embryonic diapause. Consistently, gene ontology analysis of the common genes showed that they were related to pathways expected to be involved in embryonic diapause. The present study demonstrates that ULF-EVs are important in the induction of embryonic diapause. EVs contain many other components. The lack of some of these components could explain the differential expression of genes between the let-7ginduced and in vivo dormant embryos. The differentially expressed genes may be responsible for the phenotypes that are different between the let-7induced and the in vivo embryonic diapause, such as high lactate production after let-7induced embryonic diapause. Their absence may also explain the inability of an overexpression of let-7 alone in maintaining the survival of embryos for a very long term.

The endometrium produces ULF-EVs containing proteins, mRNAs, and microRNAs that are believed to be important means of communication between the blastocysts and the endometrium (9). Let-7 containing EVs inhibited the differentiation of human embryo surrogates and reduced their attachment onto receptive endometrial epithelial cells. Dysregulation of endometrial microRNAs occurs in subfertile women (35). It is possible that an abnormal expression of embryonic diapause-related microRNAs in the endometrial-derived EVs would retard the differentiation of the implanting embryos, leading to asynchronous development between the embryos and the endometrium. Desynchronization in the development between the blastocyst and the endometrium is a cause of implantation failure (36). However, further studies are required to explore this possibility.

In conclusion, the study showed an important role of endometrial EVs in embryo dormancy, demonstrating that let-7 in EVs is a major player in the induction of embryonic diapause.

A summary of the techniques and procedures for addressing the questions raised in the report can be found in the Supplementary Materials.

The study protocol was approved by the Committee on the Use of Live Animals in Teaching and Research, The University of Hong Kong (CULATR number: 3560-15). Females of the ICR mice were mainly used. Transgenic mice carrying a DOX-inducible let-7g gene (Slet-7gLmiR-21) were generated by the injection of embryonic stem cells carrying the let-7gStem/21loop sequence (a gift from G. Daley, Harvard Stem Cell Institute, Boston, MA, USA) into ICR blastocysts. The chimeric mice generated were then bred with CD-1 females to generate germline-transmitted pups. To match the genetic background of the embryonic stem cells carrying the transgene (V6.5 mESC), the mouse line was maintained on a C57/B6 background by backcrossing more than five times.

ICR female mice aged 6 to 8 weeks were superovulated by successive intraperitoneal injections of 5 IU of pregnant mare serum gonadotropin (Sigma-Aldrich, St. Louis, USA) and 5 IU of human chorionic gonadotropin (hCG; Sigma-Aldrich) 47 to 48 hours apart and were mated with male mice. The vaginal plug was checked the day following mating. The day when a vaginal plug was seen was considered as day 1 of pregnancy. Blastocysts were collected from the uteri at 96 hours after hCG injection and cultured in M16 medium (Sigma-Aldrich) or KSOM + AA medium (Millipore, MA, USA). In normal pregnancy, blastocysts are activated by an estradiol surge in the afternoon of day 4 of pregnancy.

Delayed implanting mice were generated as reported (7). Briefly, pregnant mice were ovariectomized in the morning (8:00 a.m. to 9:00 a.m.) of day 4 followed by daily subcutaneous injection of progesterone (P4; 2 mg per mouse; Sigma-Aldrich) from days 5 to 7 (5) to maintain the delayed implantation status. Dormant blastocysts were activated by a single subcutaneous injection of E2 (25 ng per mouse; Sigma-Aldrich) into the delayed implanting mice on day 7 of pregnancy.

Female mice were mated with vasectomized males to generate pseudo-pregnant mice. Embryos were flushed out on day 4 of pregnancy and cultured as described above. Before embryo transfer, the mice were anesthetized with pentobarbital (40 mg/kg, intraperitoneally). Twenty embryos were transferred to each mouse. The implantation sites on day 5 of pregnancy were identified by intravenous injection of 0.1 ml of 1% Chicago blue dye (Sigma-Aldrich) in saline, or the numbers of live birth were recorded.

Endometrial epithelial and stromal cells were isolated after the collection of ULF. Briefly, the uteri were opened longitudinally, and the tissues were digested in trypsin (Difco, BD Biosciences, MD, USA) for 1.5 hours at 4C and then for 30 min at 37C. Dulbeccos modified Eagles medium (DMEM)/F12 medium (Sigma-Aldrich) supplemented with 10% FBS (Thermo Fisher Scientific, CA, USA) was used to stop the digestion. The epithelial cells were collected after a gentle shaking to separate the cells from the uterine tissues and were washed with phosphate-buffered saline (PBS) thrice at 300g for 10 min. The cells were then cultured briefly for 30 min to remove the contaminated stromal cells before reseeding to another culture well for experimentation. The remaining tissues were further digested by collagenase I (10 mg/ml; Invitrogen, Carlsbad, USA) and deoxyribonuclease (DNase; 5 mg/ml; Invitrogen) for 30 min with shaking at 37C. After stopping the digestion as described above, the stromal cells were shaken from the tissues. The cell pellets collected were washed by PBS thrice and filtered through a nylon mesh with pore size of 40 m (BD Falcon Co., NJ, USA) before RNA isolation. The purity of the epithelial cells and stromal cells was over 90% as determined by immunostaining, using antibodies against mouse cytokeratin (Dako, Glostrup, Denmark) and mouse CD90 (BD Biosciences, MA, USA), respectively.

Electroporation. Pre-let-7a or scrambled miRNA control (Thermo Fisher Scientific) was electroporated into day 4 blastocysts from ICR mice as described (7). Briefly, the uteri of day 4 pregnant mice were flushed with Hanks solution to obtain blastocysts, which were then transferred to prewarmed droplets of M16 medium. Pre-let-7a or scramble control was electroporated into the embryos in a flat electrode chamber (1-mm gap between electrodes; BTX Inc., San Diego, USA) in 20 l of Hepes-buffered saline (150 mM NaCl, 20 mM Hepes, Sigma-Aldrich), by two sets of three electric pulses of 1 ms at 30 V with 1-min interval between sets, and inverting polarity using the 830 Electro Square Porator (BTX Inc., San Diego, USA). Following electroporation, the embryos were cultured in KSOM + AA or M16 for experimentation. About 95% of the electroporated embryos survived the process; they showed no sign of cell lysis at 2 hours after electroporation.

Treatment with let-7enriched EV. Ishikawa cells were transfected with pre-let-7a or pre-miR scramble using Lipofectamine 2000 (Thermo Fisher Scientific). After transfection, the transfection medium was replaced by fresh MEM medium supplemented with 1% penicillin/streptomycin, 1% l-glutamine, and 10% EV-depleted FBS (Thermo Fisher Scientific, CA, USA). The spent medium after 48 hours of culture was collected for EV isolation. EVs were isolated from the spent medium with the Total Exosome Isolation Kit (Thermo Fisher Scientific) according to the manufacturers instruction. The let-7a levels in the EVs were detected by reverse transcription qPCR (RT- qPCR) (let-7a primer, Thermo Fisher Scientific). Alternatively, let-7genriched EVs were isolated from the spent medium after culture of DOX-treated endometrial epithelial cells from the let-7g transgenic mice in medium supplemented with 10% EV-depleted FBS for 48 hours. The epithelial cells with a purity of more than 90% were isolated as above. The protein concentration of the EV preparation was determined with the BCA Protein Assay Kit (Thermo Fisher Scientific) with a working concentration range of 5 to 2000 g/ml. The let-7enriched EVs at a final concentration of 100 g/ml were used for coincubation with embryos.

Pregnant mice or delayed implanting mice at 3, 6, and 24 hours after E2 reactivation were sacrificed by an overdose of pentobarbital (150 to 200 mg/kg, intraperitoneally). Their uteri were isolated, and ULF was collected by flushing the uterine lumen with 500 l of PBS. The embryos in the flushing were removed under a dissection microscopy. EVs in the ULF were isolated with the Total Exosome Isolation Kit. Briefly, the ULF was centrifuged successively at 300g for 10 min to remove cells, at 2000g for 10 min to remove dead cells, and at 20,000g for 60 min to remove cell debris and large vesicles. The pellets that formed in each centrifugation were discarded. After the last centrifugation, the total exosome isolation reagent (250-l volumes) was mixed with the supernatant overnight at 4C on a roller mixer, before the samples were centrifuged at 10,000g for 60 min at 4C. The supernatant was discarded, and the ULF-EV pellet was gently washed once with 200 l of PBS to remove residual extract buffer and resuspended in 20 l of PBS and stored at 80C. The size and purity of the isolated ULF-EVs were determined using a nanoparticle tracking analyzer (ZetaView PMX 120, Particle Metrix, Germany), electron microscopy (FEI Tecnai G2 20 Scanning TEM, FEI Co., USA), and Western blot analysis of EV-specific markers HSP70 (Abcam, Cambridge, UK), CD63 (Abcam), and TSG101 (Abcam) and negative control markers GM130 (Abcam) and calnexin (Abcam), as described (16). The average protein content of pooled ULF-EV preparations was 1.2 0.2 g per mice as determined with the BCA Protein Assay Kit (Thermo Fisher Scientific). The volume of mouse ULF on days 2 to 5 of pseudo-pregnancy is 2 to 5 l per mice (37). Therefore, the protein concentration of EV in ULF was estimated to be 240 to 600 g/ml in vivo. In this study, embryos were treated with EVs at a physiological dose of 100 g/ml.

Cell apoptosis was examined with the In Situ Cell Death Detection Kit (Roche Diagnostics GmbH, Mannheim, Germany) according to the manufacturers instruction. Briefly, embryos were fixed in 4% paraformaldehyde for 15 min at room temperature, permeabilized with 0.1% Triton X-100 in Dulbeccos PBS (DPBS) for 30 min, and then incubated with TUNEL reaction mixture containing 5 l of enzyme solution and 45 l of label solution at 37C for 60 min. The embryos were washed three times with PBS. Their nuclei were stained with propidium iodide (Sigma-Aldrich) for 5 min before the embryos were mounted on microscope slides for examination under a fluorescence-inverted microscope (TE300; Nikon, Japan).

The method used was a miniaturized version of conventional enzymatic methods, which rely on the detection of ultraviolet (UV)excited NADH (reduced form of nicotinamide adenine dinucleotide) and NADPH (reduced form of nicotinamide adenine dinucleotide phosphate) in enzyme coupled reactions (38). Instead of having the enzymatic reactions conducted in cuvettes, they were done in 20-l droplets on a petri dish. The specific enzyme cocktails for the metabolite studied were as follows: glucose cocktail: 42 mM EPP S (4-(2-Hydroxyethyl)-1-piperazinepropanesulfonic acid) buffer (pH 8.0), 42 M dithiothreitol, 3 mM MgSO47H2O, 0.42 mM ATP, 1.2 mM NADP, and hexokinase (14 U/ml)/glucose-6-phosphate dehydrogenase (7 U/ml; Roche Applied Science); pyruvate cocktail: 63 mM EPP buffer (pH 8.0), 0.1 mM NADH, and l-lactate dehydrogenase (75 U/ml; Roche Applied Science); lactate cocktail: 0.45 M glycine/0.73 M hydrazine buffer, 4.5 mM NAD, and l-lactate dehydrogenase (69 U/ml; Roche Applied Science).

The cocktail droplet (2 l) was mixed with 18 l of spent culture media. Following a 3-min incubation at room temperature, 5 l of the medium was transferred to a homemade chamber with a chamber depth of 1 mm, and its fluorescence intensity was determined under a fluorescence microscope. The fluorescence signal relative to the background was determined by the pixel intensities using the Image-Pro Plus 6.0 software (Media Cybernetics Inc., Silver Spring, MD, USA). The background signal was estimated using a method described previously (39). The changes in fluorescence were converted to changes in the concentration based on standard curves performed on the same day with known concentrations of the appropriate substrates.

DNA synthesis was determined by the EdU incorporation assay (Thermo Fisher Scientific) according to the manufacturers instruction. Briefly, embryos were cultured in KSOM medium containing 10 M EdU for 30 min before washing with PBS. After the removal of the zona pellucida, the embryos were fixed in methanol at 20C for 20 min and permeated in PBS containing 1% bovine serum albumin (BSA) and 0.5% Triton X-100. The incorporated EdU was detected by incubation with 1 mM CuSO4 and 100 M fluorescent azide for 30 min. The staining mixture was prepared fresh each time. The embryos were washed three times with PBS containing 0.05% Tween 20 before the fluorescence signal was visualized under a confocal microscope (Carl Zeiss LSM 700, Zeiss, Germany).

The embryos were washed with M2 medium (Sigma-Aldrich) and fixed in 4% paraformaldehyde for 15 min at room temperature. They were permeabilized with 0.1% Triton X-100 in DPBS for 30 min and incubated for 1 hour in DPBS containing 1% BSA at room temperature before incubation with antibodies against C-MYC, pAKT, pRpS6, p4EBP1 (Abcam, UK), or Ki67 (Santa Cruz Biotechnology Inc., Santa Cruz, CA) at 4C overnight followed by incubation with secondary antibody [FITC-labeled anti-goat immunoglobulin G (IgG)] for 1 hour at 37C. Nuclei were stained with 4,6-diamidino-2-phenylindole (DAPI) (5 g/ml; Sigma-Aldrich) or propidium iodide (1 g/ml) for 5 min. Last, the embryos were rinsed in DPBS to remove excess reagents and examined under a confocal microscope.

Proteins were extracted from the JEG-3 cells and the uterine epithelial cells with radioimmunoprecipitation assay (RIPA) lysis buffer supplemented with protein inhibitor and phosphatase inhibitors (Sigma-Aldrich). The concentration of proteins in the extract was measured using the BCA Kit (Thermo Fisher Scientific). The protein extracts were separated on 10% SDSpolyacrylamide gel electrophoresis, transferred onto polyvinylidene difluoride membranes (Merck Millipore, Germany), and probed with antibodies against c-myc (Cell Signaling Technology, MA, USA), ODC1, SMS, pAKT, p4EBP1, pRpS6, and the total protein of AKT, 4EBP1, and RpS6 (Cell Signaling Technology). -Actin (Sigma-Aldrich) was used as the internal control. The membranes were incubated with the WesternBright ECL Kit (Advansta, CA, USA) and exposed to x-ray films.

Embryos at the same developmental stage were randomly pooled into three groups with five embryos per group. Total RNA was extracted from each group in 0.5 l of 2 M guanidine isothiocyanate (Sigma-Aldrich) buffer at room temperature for 5 min. Complete lysis of the embryos in the buffer was confirmed under a microscope. The samples were diluted to 5 l with double-distilled water and were used directly for the multiplex microRNA assays.

To amplify the precursor-microRNA, the forward and reverse primers were designed to anneal to the stem portion of the hairpin. The forward primer was designed for a sequence that crossed the stem and the loop of the precursor of Slet-7gLmiR-21 so that only its stem, but not that of the pre-let-7g, was amplified. The sequences of the primers used for pre-S7gL21 were as follows: forward: 5-GTAGTAGTTTGTACAGTTCTGT-3; reverse: 5-TAAATCCTGGCAAGGC-3; probe: 5-CTGTACAGTCCATGAGATT-3; Rt reverse: 5-TAAATCCTGGCAAGGCA-3.

Embryos were lysed in 5% NP-40 to release the total RNA. RT primer annealing was performed at 85C for 5 min by adding 1 pmol of RT reverse primer to 1 g of the above RNA. Then, the samples were placed immediately in ice to avoid the formation of stem-and-loop structure. RT was performed at 45C for 60 min and at 85C for 5 min and then kept at 4C in a thermal cycler (T100 Thermal Cycler, Bio-Rad, CA, USA). RT-qPCR was performed with the 7500 Real-Time PCR System (Thermo Fisher Scientific). The program was a 10-min cycle at 95C, followed by 45 cycles of 15 s at 95C and 60 s at 60C.

Microarray (GeneChipTM Mouse Gene 2.0 ST) was used to study the effect of let-7 overexpression on the transcriptome of the treated blastocysts. For each sample, RNA was collected from 10 blastocysts. RNA extraction, amplification, and purification were performed according to Kurimoto et al. (40). The complementary DNAs (cDNAs) from day 7 dormant blastocysts, D4-act, and DOX-induced blastocysts were hybridized to the GeneChipTM Mouse Gene 2.0 ST array in duplicates (Affymetrix, CA, USA). All cDNA hybridizations were performed by the Centre for Genomic Sciences, The University of Hong Kong. The microarray data have been deposited to the Gene Expression Omnibus (GSE141900). The data were analyzed using Partek Genomics Suite 6.6 (St. Louis, MO, USA). The expression matrix was further subjected to the R package linear models for microarray data (41) for identifying the differentially expressed genes. Principal components were computed and plotted with the R packages FactoMineR and factoextra. Heatmaps were plotted with the R package gplots using z scores calculated for each gene across different samples. Biological process analysis was performed by DAVID (v6.8) (42).

To obtain the DNA template for in vitro transcription, the pcDNA 3.1_cMyc plasmid containing the coding DNA sequence was PCR-amplified using the following primers: 5-TAATACGACTCACTATAGATGCCCCTCAACGTGAAC-3 (with T7 polymerase promoter) and 5-TTATGCACCAGAGTTTCGAAGC-3. The product was purified using the GeneJET PCR Purification Kit (Thermo Fisher Scientific). The MEGAscript T7 ULTRA Transcription Kit (Thermo Fisher Scientific) was used for in vitro transcription from DNA to mRNA according to the manufacturers instructions. Briefly, DNA template was recovered at a final concentration of 1 g/l. Transcription reaction was performed by incubation at 37C for 4 hours. TURBO DNase (1 l) was added into the reaction and incubated for 15 min at 37C before the addition of the tailing reagents for poly(A) tailing. Last, RNA was recovered using phenol:chloroform extraction and isopropanol precipitation. The recovered RNA was then quantified and stored at 80C and was ready for transfection.

The mouse genomic DNA was extracted from the ICR mouse liver using a DNA extraction kit (Thermo Fisher Scientific) according to the manufacturers protocol. The 3UTR of Rictor was amplified with the Not I and Xho I digestion sites by the Phusion High Fidelity DNA Polymerase (New England Biolabs, Beverly, MA, USA). The PCR products were first purified with the GeneJET PCR Purification Kit (Thermo Fisher Scientific), digested with the Not I and Xho I enzymes (New England Biolabs), and purified with the GeneJET PCR Purification Kit again. The purified PCR products were ligated with the Not I and Xho Idigested psiCHECK-2 vector (Promega, WI, USA). Lipofectamine 2000 transfection reagent (Thermo Fisher Scientific) was used to cotransfect 1 g of the WT and mutant (Mut) reporter constructs with 5 nM let-7a mimic (Thermo Fisher Scientific) into a monolayer of JEG-3 cells at 70% confluence in Opti-MEM (Thermo Fisher Scientific). At 24 hours after transfection, the cells were lysed in 100 l of 1 passive lysis buffer (Promega). The luciferase assays were performed using a luciferase assay kit (Promega) according to the manufacturers protocol and were measured using a luminometer (GloMax 96 Microplate Luminometer, Promega). Renilla luciferase was used for normalization.

ChIP analysis was performed using the Pierce Chromatin Prep Module (Thermo Fisher Scientific, 26158) according to the manufacturers instruction. Uterine epithelium cells from delayed implanting and activated mice were isolated as described above. Formaldehyde was used to cross-link DNA and its interacting proteins in the cells. The cells were then lysed in the Lysis Buffer on ice for 10 min and centrifuged. The supernatant was discarded, while the nuclei were resuspended in the MNase Digestion Buffer. Micrococcal nuclease was added to digest the chromatin. Immunoprecipitation was performed with the ChIP-grade c-Myc antibody (Cell Signaling Technology) at a dilution of 1:100. Proteinase K was used to disrupt the cross-links between the DNA and proteins. The DNA was then purified using the PCR Cleanup Extraction Kit (Thermo Fisher Scientific), after which quantitative RT-PCR was performed with the SYBR Green Master Mix (Thermo Fisher Scientific) using the following ChIP primer sequences (Table 1).

The data obtained were normalized to the input [fold differences = 2(Ct sample Ct input)].

Blastocysts were incubated for 10 min at 37C in an atmosphere of 5% CO2 in 40-l microdrops of M16 containing Alexa Fluor 488labeled EGF (2 g/ml; Thermo Fisher Scientific). Unlabeled EGF peptide at a concentration of 20 g/ml was used as control for nonspecific binding of the labeled peptide. After termination of the incubation, blastocysts were washed in medium and fixed in 4% paraformaldehyde in PBS for 15 min at 4C. Z-stack images of fixed embryos were captured with a confocal fluorescence microscope (Carl Zeiss LSM700, Germany). For quantification of the EGF binding, the images of the embryos were analyzed with the ImageJ software (1.52p, USA). The average fluorescence intensity was calculated. The data presented were the averages of the fluorescence intensity from at least three embryos.

BAP-EB spheroids were generated from hESCs as described (23). Briefly, hESCs (VAL3, Spanish Stem Cell Bank, Spain) were digested to single cells with accutase (Thermo Fisher Scientific, Waltham, USA) and aggregated in AggreWellTM400 (STEMCELL Technologies Inc., Canada) in mTeSRTM1 medium (STEMCELL Technologies Inc., Canada) for 24 hours before the induction of trophoblast differentiation in BAP medium (mouse embryonic fibroblastconditioned medium supplemented with BMP4 (10 ng/ml; R&D Systems, Minneapolis, USA), 1 M A83-01 (Stemgent, San Diego, USA), and 0.1 M PD173074 (Stemgent). The medium was changed daily during a 96-hour differentiation.

Control EVs or let7-enriched EVs were added to the BAP-EB culture at 48-hour pid. For the attachment assay, BAP-EB at 72-hour pid was transferred onto a confluent monolayer of Ishikawa cells and cocultured for 3 hours. Nonadherent spheroids were removed, and the percentage of attached BAP-EB was determined. For the gene expression analyses, BAP-EB at 0 and 48 hours before EV treatments and BAP-EB at 96 hours after EV treatments were collected and subjected to total RNA extraction and real-time quantification of marker expressions, as described (23).

Human embryos were obtained from infertile couples attending the assisted reproduction clinics at the Department of Obstetrics and Gynecology, General Hospital of Chinese Peoples Liberation Army and the Center for Reproductive Medicine, The Third Affiliated Hospital, Sun Yat-Sen University. The Institutional Review Board of the Hospital approved the project (S2017-095-01), and written consent was obtained from each donor. The embryos were donated because the donor couples had completed their family (N = 17), or the embryos were chromosomal abnormal as determined by preimplantation genetic testing for aneuploidy (N = 4). The donated embryos were cryopreserved on day 5 before experimentation.

EVs were obtained from endometrial cells of let-7g transgenic mice after treatment with DOX in DMEM/F12 medium supplemented with EV-free FBS for 4 days. Control EVs were obtained from cells without DOX treatment. On the day of experimentation, the donated blastocysts were thawed and cultured in G2 medium (Vitrolife, Sweden) supplemented with let-7genrched EVs or control EVs until they were morphologically not viable.

All the results are shown as means SEM. All the data were analyzed using one-way analysis of variance (ANOVA). A P value of less than 0.05 was considered statistically significant.

Acknowledgments: Funding: The project is supported by a grant from the National Natural Science Foundation of China (NSFC 31471398); a grant from the Research Grant Council (GRF 17107915), Hong Kong; and a National Key Basic Research Development Program (973 Program) from the Ministry of Science and Technology of the Peoples Republic of China (MOST 2018YFC1004402) to W.S.B.Y. Author contributions: Conceptualization: W.S.B.Y., W.M.L., and R.R.C.; conduct of the experiments: W.M.L., R.R.C., Z.R.N., M.Y.M., T.L., P.C.C., and R.T.P.; data analysis: W.S.B.Y., Y.L.L., W.M.L., and A.C.C.; writing (original draft): W.S.B.Y. and W.M.L.; writing (review and editing): all authors. Competing interests: The authors declare that they have no competing interests. Data and materials availability: All data needed to evaluate the conclusions in the paper are present in the paper and/or the Supplementary Materials. The microarray data have been deposited to the Gene Expression Omnibus. Additional data available from W.S.B.Y. upon request.

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Mini-Guts Could Offer Treatment Hope for Children With Intestinal Failure – Technology Networks

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Pioneering scientists at the Francis Crick Institute, Great Ormond Street Hospital (GOSH) and UCL Great Ormond Street Institute of Child Health (ICH) have grown human intestinal grafts using stem cells from patient tissue that could one day lead to personalised transplants for children with intestinal failure, according to a study published in Nature Medicine on Monday 7th September.

Children with intestinal failure cannot absorb the nutrients that are essential for their overall health and development. This may be due to a disease or injury to their small intestine.

In these cases, children can be fed intravenously via a process called parenteral nutrition, however this is associated with severe complications such as line infections and liver failure. If complications arise or in severe cases these children may need a transplant. However, there is a shortage of suitable donor organs and problems can arise after surgery, such as the body rejecting the transplant.

In their proof-of-concept study, the research team showed how intestinal stem cells and small intestinal or colonic tissue taken from patients can be used to grow the important inner layer of small intestine in the laboratory with the capacity to digest and absorb peptides and digest sucrose in food.

This is the first step in efforts to engineer all the layers of the intestine for transplantation. The researchers hope that one day, laboratory grown organs could offer a safe and longer-lasting alternative to traditional donor transplants.

"It's urgent that we find new ways to care for children without a working intestine because, as they grow older, complications from parental nutrition can arise," says Dr Vivian Li, senior author and group leader of the Stem Cell and Cancer Biology Laboratory at the Crick.

"We've set out a process to grow one layer of intestine in the laboratory, moving us a step closer to being able to offer these patients a form of regenerative medicine, which uses materials created from their own tissue. This would reduce some of the risks that transplant patients face, such as their immune system attacking the transplant."

The researchers took small biopsies of intestine from 12 children who either had intestinal failure or were at risk of developing the condition. In the lab, they then stimulated the biopsy cells to grow into "mini-guts", also known as intestinal organoids, generating over 10 million intestinal stem cells from each patient over the course of 4 weeks.

The researchers also collected small intestine and colon tissue, that would otherwise have been discarded, from other children undergoing essential surgery to remove parts of their gut. Using laboratory techniques, cells were removed from these tissues leaving behind a skeleton structure which formed scaffolds.

The researchers placed the "mini-guts" onto these scaffolds, where they grew on this structure to form a living graft. Due to specific culture conditions, the stem cells changed into many of the different types of cells that exist in the small intestine. The grafts were able to digest and absorb peptides, the building blocks of proteins, as well as digest sucrose into glucose sugars.

"Although this research is in the lab right now, we're concentrating on making this a realistic and safe treatment option," explains senior author NIHR Professor Paolo De Coppi, Consultant Paediatric Surgeon at GOSH and Head of Surgery, Stem Cells & Regenerative Medicine Section at the UCL Great Ormond Street Institute of Child Health (ICH).

"What's significant here is we've shown that scaffolds can be created using tissue from the colon, not only tissue from the small intestine. In practice, it is often easier to obtain tissue from the colon, so this could make the approach much more feasible. It's an important step forward in regenerative medicine and we're optimistic about what this means for patients, but more research lies ahead before we can safely and effectively translate this approach to treatment."

As well as proving that biopsies taken from children could be used to grow functioning intestinal grafts, the researchers also demonstrated that the grafts survive and mature when transplanted into mice.

"By applying our basic science knowledge of intestinal stem cell biology, we have developed a time efficient and clinically relevant method for rebuilding human small intestine grafts for transplantation," says Laween Meran, lead author, Gastroenterology Registrar and Clinical Research Training Fellow at the Stem Cell and Cancer Biology Laboratory at the Crick and the ICH.

"Now that we've shown the grafts are successful on a small scale, the next crucial steps will be to start growing the other layers of the intestine such as muscle and blood vessels, whilst also scaling up our methods to create viable grafts relevant to individual patient needs".

Reference:Meran, L., Massie, I., Campinoti, S. et al. Engineering transplantable jejunal mucosal grafts using patient-derived organoids from children with intestinal failure. Nature Medicine.2020. doi.org/10.1038/s41591-020-1024-z

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Mini-Guts Could Offer Treatment Hope for Children With Intestinal Failure - Technology Networks

Balzarotti Breaks Down New Developments in the Expanding DLBCL Paradigm – OncLive

In recent years, rituximab (Rituxan), cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) has been the standard treatment for patients with diffuse large B-cell lymphoma (DLBCL), according to Monica Balzarotti, MD, but new approaches such as selinexor (Xpovio), bispecific antibodies, and CAR T-cell therapies are expanding the armamentarium.

Notably, about 20% of patients with DLBCL have refractory disease. Some patients are not suitable for high-dose consolidation with autologous stem cell transplantation (ASCT) because there is no treatment that can guarantee a longer remission, Balzarotti explained. Whatever you usechemotherapy, such as gemcitabine or oxaliplatin, polatuzumab vedotin (Polivy)/bendamustine, or even new experimental drugs[we are not able to obtain] longer remissions. Looking forward, we must determine whether we can consolidate and maintain responses.

In an interview with OncLive, Balzarotti, a hematologist in the Department of Hematology at the Institute for Research, Hospitalization, and Health Care (IRCCS) Humanitas Research Hospital, discussedthe current treatment landscape of DLBCL, including the recent FDA approval of selinexor for the treatment of adult patients with relapsed/refractory disease.

OncLive: Could you discuss the current treatment landscape for DLBCL?

Balzarotti: In the first-line treatment of [patients with] DLBCL, R-CHOP is still the golden standard because no other regimen has been able to demonstrate superiority [over this approach]. However, there is some hope regarding the use of BTK inhibition in younger patients. The PHOENIX trial demonstrated the superiority of R-CHOP plus ibrutinib (Imbruvica) in younger patients; however, the primary end point concerned older patients. Thus, we dont have a definitive [takeaway] from this trial. Another trial will examine acalabrutinib (Calquence) in younger patients that might help us to answer this question.

At relapse, [we use] high-dose consolidation with ASCT; this approach is reserved for younger patients aged up to 70 years. This strategy can cure about 50% of patients with relapsed disease, even the patients that experience relapse later on. In the case of primary refractory disease, or patients with very early progressive disease, even chemotherapy and ASCT is not enough to guarantee a high percentage of cure. Under 20% of these patients have the probability to survive 1 year.

Now, we have CAR T-cell therapy and we are very hopeful. This is a new strategy that is very promising, but I don't believe it will be able to cure all patients with relapsed/refractory disease. Thus, we need other drugs and more knowledge of the biology of the disease.

Could you expand on some of the later-line treatment approaches?

We have several therapies, many of which are concentrated [for use] in the subgroup of patients who are not suitable for ASCT. The majority of patients with DLBCL are not eligible for ASCT, because this disease mainly effects the elderly. Oftentimes, elderly patients do not receive an optimal treatment in the first-line setting. Sometimes they receive a reduced dose of R-CHOP and then they relapse, or they have a comorbidity that does not allow for them to receive full-dose chemotherapy.

As such, several therapies have been developed. One such therapy is lenalidomide (Revlimid), which can result in a 30% complete remission (CR) rate. This is higher in patient with the Germinal center B-cell like subtype. This is the only drug that has been approved for use worldwide.

Another agent is polatuzumab vedotin, which is a CD79b-directed antibody-drug conjugate (ADC); this drug has been approved for use in combination with bendamustinein the United States and it [will likely] be approved in Europe and Italy in the future. However, this has been delayed because of the novel coronavirus 2019 pandemic.

We also have several other drugs and strategies, such as the bispecific antibodies, ADCs, and monoclonal antibodies, such as cabozantinib (Cabometyx). We also have selinexor, CAR T-cell therapy, ASCT, and maybe some other drugs that are still in earlier phases of experimentation. We have many treatments, but we still need to learn how best to use them and how to sequence them appropriately.

What are the big unmet needs that still need to be addressed?

Oftentimes, DLBCL is not a good candidate for maintenance therapy because it does not have a molecular marker such BCL-2 which is used in follicular lymphoma, chronic lymphocytic leukemia, and mantle cell lymphoma. However, when evaluating circulating tumor DNA and the identification of newer molecular markers, we may be able to proceed in that way. We have small experience with the consolidation and maintenance treatment, for example, with lenalidomide and other drugs, which is encouraging.

What ongoing studies are you most excited about?

In the first-line setting, the most highly anticipated results are those from the POLARIX trial, which compared R-CHOP with R-CHOP plus polatuzumab vedotin. We also anticipate results from another trial, which is comparing ASCT with CAR T cells in the setting of refractory disease. Other trials are in earlier stages and are currently accruing.

Could you speak to the recent FDA approval of selinexor in DLBCL?

Selinexor has shown encouraging activity with a high rate of both CR and overall remission. In the published trial, there were longer durations of complete and partial remissions. The fact that remission can be maintained is important, especially for the maintenance therapy that I mentioned earlier.

On the other hand, I know that selinexor is not an easy drug to manage, especially because of the high rate of nausea and vomiting. There are very aggressive protocols to contain these toxicities [but it] is a bit worrying for the European investigators; we are not used to administering these drugs. There is an agent in the United States that is used to help control these symptoms.

However, I believe this could potentially be problematic because, when prescribing a drug for a long period of time, the adverse effects may hinder a patients quality of life. We need to ensure that patients are taking the drug as directed.

Where should future research efforts be focused with this agent?

Its important to learn how to manage selinexor [better]. When there is a new drug, you read the literature to better understand the activity of the drug, but only for a very selective group of patients. Then, there is the real world. We have real-world data on CAR T cells and its interesting to think about; this is better. We are now waiting for the real-world [data] of selinexor [to see what can be learned].

What is your take-home message to your colleagues treating patients with DLBCL?

The most important message is that patients with relapsed disease should be managed by the referral center, not because the conditions of the center are better, but because they usually have new drugs, strategies, and ways to approach patients.

In one center they may have selinexor, in another they may have ADCs, and another may have CAR T-cell therapy. You need a center or a collaborative group where you can find all of these strategies because this will help you choose the best therapy because you will have a better understanding of the risks and characteristics of the drug. Sometimes these strategies can be used in segments.

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Balzarotti Breaks Down New Developments in the Expanding DLBCL Paradigm - OncLive

Application of Immunotherapy to $7 Billion Lower Back Pain Market Patented by Creative Medical Technology Holdings – BioSpace

PHOENIX, Sept. 8, 2020 /PRNewswire/ --(OTC CELZ) -- Creative Medical Technology Holdings Inc. announced today receipt of a Notice of Allowance from the United States Patent and Trademark Office for its patent application, "Perispinal Perfusion by Administration of T Regulatory Cells Alone or in Combination with Angiogenic Cell Therapies."

The patent covers the use of activated T regulatory cells for inducing an increase in blood circulation in areas surrounding the disc of patients with lower back pain. It is believed that a significant proportion of patients suffering from lower back pain have abnormally poor circulation, which does not effectively remove waste products and irritants. Restoration of circulation in the lower back is associated with reduction of pain.

The Company acquired a previously granted US Patent # 9,598,673 covering use of various types of stem cells, autologous and allogeneic, for treating lower back pain. The Company has completed an autologous cell therapy pilot study in the area of lower back pain utilizing this patented technology and is currently in the process of assembling data for publication.

"Creative Medical Technology Holdings is developing a critical mass of issued intellectual property covering multiple cell therapy treatments of lower back pain as well as other indications," said Timothy Warbington, President and CEO of the Company. "Testimony to the size of the lower back pain market is the $1 Billion Mesoblast-Grunenthal deal for a pre-review cell therapy product1. We are enthusiastic to add this new therapy to our expanding portfolio of rapid-to-commercialize cellular therapies."

Creative Medical Technology Holdings has previously commercialized its CaverStemR technology involving personalized bone marrow cellular therapy for erectile dysfunction. This technology is covered by issued patent # 8,372,797 and a clinical trial demonstrating safety with signals of efficacy published in the peer-reviewed literature2.

"Immunotherapy is one segment of the biotechnology industry that is expanding at an exponential rate," said Donald Dickerson, CFO of the Company. "The recent Nobel Prize in the area of Immunotherapy of Cancer, as well as the current valuations of immunotherapy companies, validates the approaches that we have been developing, and now patenting. Essentially our approach is to use stem cells, or immunotherapy to enable the body to heal itself."

"The Company welcomes the biotechnology/life sciences community and key opinion leaders to contact us to discuss potential collaboration on our patented technologies in this amazing space," Mr. Warbington said further.

About Creative Medical Technology Holdings

Creative Medical Technology Holdings, Inc. is a commercial stage biotechnology company specializing in stem cell technology in the fields of urology, neurology and orthopedics and trades on the OTC under the ticker symbol CELZ. For further information about the company, please visit http://www.creativemedicaltechnology.com.

Forward Looking Statements

OTC Markets has not reviewed and does not accept responsibility for the adequacy or accuracy of this release. This news release may contain forward-looking statements including but not limited to comments regarding the timing and content of upcoming clinical trials and laboratory results, marketing efforts, funding, etc. Forward-looking statements address future events and conditions and, therefore, involve inherent risks and uncertainties. Actual results may differ materially from those currently anticipated in such statements. See the periodic and other reports filed by Creative Medical Technology Holdings, Inc. with the Securities and Exchange Commission and available on the Commission's website at http://www.sec.gov.

Creativemedicaltechnology.com http://www.StemSpine.com http://www.Caverstem.com http://www.Femcelz.com

1https://www.biopharma-reporter.com/Article/2019/09/12/Gruenenthal-partners-with-Mesoblast-for-back-pain-cell-therapy#:~:text=Gr%C3%BCnenthal%20agrees%20deal%20with%20Mesoblast,disease%20in%20previously%20treated%20patients.

2https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6958721/

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Stem Cell Alopecia Treatment Market is Thriving Worldwide 2020 | Trends, Growth and Profit Analysis, Forecast by 2027 – The Daily Chronicle

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Global Stem Cell Alopecia Treatment Market, By Indication

Male Pattern Baldness Female Pattern Baldness Others

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Stem Cell Therapy Market Research, Technology, Demand, Analysis, Services, Type and Global Industry Forecast 2025 – The Daily Chronicle

Introduction & Scope: Global Stem Cell Therapy Market

Global Stem Cell Therapy market research report presentation demonstrates and presents an easily understandable market depiction, lending crucial insights on market size, market share as well as latest market developments and notable trends that collectively harness growth in the global Stem Cell Therapy market.

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According to research inputs, this global Stem Cell Therapy market is also likely to register a thumping growth of USD xx million in 2020 and is further anticipated to reach over xx million USD by the end of 2027, clocking at a steady CAGR of xx% through the forecast span, 2020-27. Expert opinion also suggests that the global Stem Cell Therapy market is likely to tread on the road to recovery in the foreseeable future.

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Adipose Tissue-Derived Mesenchymal SCs Bone Marrow-Derived Mesenchymal SCs Embryonic SCs Other Sources

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Musculoskeletal Disorders Wounds & Injuries Cardiovascular Diseases Gastrointestinal Diseases Immune System Diseases Other Applications

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Study on Table of Contents: Stem Cell Therapy Market Overview, Scope, Status and Prospect (2015-2020) covering COVID-19 Pandemic. Global Stem Cell Therapy Market Competition by Manufacturers Global Stem Cell Therapy Capacity, Production, Revenue (Value) by Region (2015-2020) Global Stem Cell Therapy Supply (Production), Consumption, Export, Import by Region (2015-2020) Global Stem Cell Therapy Production, Revenue (Value), Price Trend by Type Global Stem Cell Therapy Manufacturers Profiles/Analysis Stem Cell Therapy Manufacturing Cost Analysis Industrial Chain, Sourcing Strategy and Downstream Buyers Marketing Strategy Analysis, Distributors/Traders Global Stem Cell Therapy Market Effect Factors Analysis and Forecast (2020-2025)

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FDA Grants Breakthrough Therapy Designation to Plinabulin for Chemotherapy-Induced Neutropenia Indication – Cancer Network

The FDA granted breakthrough therapy designation to BeyondSprings first-in-class agent plinabulin for the chemotherapy-induced neutropenia (CIN) indication, according to a BeyondSpring press release.

Data for the full PROTECTIVE-2 phase 3 study is expected for the fourth quarter of 2020 and the company will file a new drug application (NDA) with the FDA by the end of 2020.

"Receipt of breakthrough therapy sesignation from the FDA acknowledges both the significant unmet need among patients with CIN and the highly encouraging clinical results generated by Plinabulin, said Douglas Blayney, MD, global principal investigator for plinabulins CIN studies, in a press release.

The designation was granted based on positive interim analysis data from the phase 3 PROTECTIVE-2 study. The data found that plinabulin combined with pegfilgrastim (Neulasta) was significantly better than pegfilgrastim alone in achieving the primary end point (p < 0.01).

More, there was a well-tolerated safety profile and fewer grade 4 adverse events with plinabulin combined with pegfilgrastim compared to pegfilgrastim alone. The primary end point of the research was the prevention of severe neutropenia.

This should expedite plinabulins move into the clinic, which is beneficial for patients, said Blayney. The currently approved CIN prevention agents are all G-CSF-based and not available to all patients. Even with the use of G-CSFs, over 80 percent of cancer patients undergoing chemotherapy may still experience Grade 4 neutropenia, which could lead to severe infection, hospitalization and even death. Thus, CIN still represents an unmet medical need.

The results from the study were further strengthened by other CIN studies from BeyondSpring confirming early onset action in week 1 with protecting neutrophils in various cancer types and various chemotherapies, which is complementary to Week 2 neutrophil protection with G-CSFs.

For patients undergoing treatment involving the destruction of white blood cells, CIN is a common side effect. For patients with grade 4 neutropenia, there exists an abnormally low concentration of neutrophils, which could lead to infection, hospitalization, or death.

Plinabulin is a differentiated immune and stem cell modulator in the late stages of clinical development to increase patient overall survival and alleviate CIN. The benefits of plinabulin are associated with its effect as a potent antigen-presenting cell (APC) inducer and T-cell activation.

"The clinical profile Plinabulin has shown truly represents a breakthrough in the CIN space since G-CSFs," Ramon Mohanlal, MD, PhD, MBA, chief medical officer and executive vice president of research and development, at BeyondSpring, said in a press release. We look forward to continuing to work with the FDA as we advance the development of Plinabulin to address this urgent medical need.

Reference:

BeyondSpring Receives Breakthrough Therapy Designations from Both U.S. FDA and China NMPA for Plinabulin in Chemotherapy-Induced Neutropenia Indication [news release]. New York, New York. Published September 8, 2020.https://www.globenewswire.com/news-release/2020/09/08/2089925/0/en/BeyondSpring-Receives-Breakthrough-Therapy-Designations-from-Both-U-S-FDA-and-China-NMPA-for-Plinabulin-in-Chemotherapy-Induced-Neutropenia-Indication.html. Accessed September 8, 2020.

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FDA Grants Breakthrough Therapy Designation to Plinabulin for Chemotherapy-Induced Neutropenia Indication - Cancer Network

Automation needed to truly scale cell therapies, Lonza – Bioprocess Insider – BioProcess Insider

Lonza says it has achieved a significant milestone with the first patient being treated with a CD19 CAR-T cell immunotherapy made using its automated point-of-care Cocoon technology.

In March 2019, Swiss CDMO Lonza entered a partnership with Israels Sheba Medical Center to provide automated and closed CAR-T manufacturing using its point-of-care (POC) Cocoon cell therapy manufacturing platform.

This week, Sheba and Lonza announced the first patient has been dosed with a CD19 CAR-T cell immunotherapy made using the technology in a phase II cancer trial.

Image: iStock/Duncan_Andison

Shebas ongoing immunotherapy program has already treated 100 oncology patients with its CAR-T CD19 therapy with good clinical success. The therapy is currently manufactured at the point-of-care using an open, clinically validated manual process. However, this first patient to be treated with a Cocoon-made CAR-T is significant as it demonstrates the potential of the Lonzas system in scaling up the manufacture of cell therapies, Eytan Abraham, head of Personalized Medicine at Lonza, said.

The ultimate goal of this, as well as other cell and gene therapies, is to treat large patient populations, he told Bioprocess Insider. The Cocoon platform represents an ideal solution for Sheba, solving the shortage of resources e.g., manpower, clean room space and manufacturing bandwidth.

A further ten patients will be tested with the Cocoon-manufactured CAR-T candidate, he added, before the results are reviewed by the Israeli Ministry of Health.

Assuming this goes well, the intention is that Sheba transitions to using the Cocoon for all CAR-T manufacturing, he continued, though noted that with a process change for any candidate being investigated in an ongoing clinical trial Sheba will need to show product comparability and receive approval from the Israeli Ministry of Health.

A full clinical comparability study confirmed that the product produced in the current manual process is indeed comparable to the product produced in the Cocoon platform.

Developed by Octane Biotech, Lonza began working with the system in 2015 to help develop the platform for autologous cell therapy manufacturing. The automated and closed platform is a single system that can be used for a variety of different autologous cell therapy protocols, including CAR-T, but also tumor-infiltrating lymphocytes (TILs) and Mesenchymal stem cells (MSCs).

In October 2018, Lonza acquired an 80% stake in Octane and according to Abraham has significantly advanced the system since.

The main activities have been improving the systems robustness and readiness for commercial sale and clinical manufacturing. This included full qualification of the system, passing all regulatory, safety and quality requirements, scaling up manufacturing, obtaining EU CE marking as well as DMF submission to the FDA, and much more.

Now that the system is launched and proven, we are hard at work, further developing it and enabling additional key capabilities, which will make it even more compelling and efficient. These include capabilities such as adding integrated magnetic cell separation and integrated cell electroporation (using the Lonza Nucleofector system).

Autologous CAR-T therapies such as Novartis Kymriah (tisagenlecleucel) and Gilead/Kites Yescarta (axicabtagene ciloleucel), both of which have been commercialized come at a cost. The personalized nature of the therapy gives rise to the adage the product is the process which, roughly translated, means scaling up manufacturing is difficult if not impossible and the cost is high.

Thus, the only way to truly scale cell therapy manufacturing is automation, said Abraham. The scalability of cell therapy manufacturing will become be critical as more cell therapies continue to reach late phase clinical trials and are commercialized.

He continued: Adding more Cocoon systems will not require much additional footprint thanks to the Cocoon tree, which allows to array multiple cocoons on a central axis. Up to 10 individual processes will be able to be run within approximately 1 m2.

On top of Sheba, other cell therapy developers have therefore turned to Lonza and its Cocoon technology to address this problem. Lonza inked a research collaboration with Stanford University School of Medicine, Fred Hutchinson Cancer Research Center, and Parker Institute for Cancer Immunotherapy earlier this year. And in March, Triumvira Immunologic announced a deal to utilize the Cocoon system, with Triumvira CEO Paul Lammers stating at the time that it is critical to leverage innovative technology to automate manufacturing processes to improve consistency, accelerate logistics, reduce footprint, and reduce cost.

Abraham added Lonza is working with other undisclosed partners to place their patient scale cell therapies into the Cocoon system for clinical manufacturing and expects to enter many more Cocoon collaborations whether with a centralized or decentralized manufacturing model.

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Automation needed to truly scale cell therapies, Lonza - Bioprocess Insider - BioProcess Insider

Using Organoids in the Study of Infectious Diseases – Technology Networks

Organoid cell culture has transformed cell-based assays in drug discovery and basic biology by conferring physiologic relevance to in vitro cell-based biological models. When provided with a suitable growth environment, including appropriate cultureware, growth factors, extracellular matrix, nutrients, and culture media, organ-derived progenitor cells harvested from patients grow and assemble into three-dimensional structures organoids which incorporate all cell types normally found in the original tissue, and allow physical and chemical interactions between and among cells. By providing greater physiologic relevance and a species- or patient-specific test platform, organoids overcome many limitations of conventional 2D cultures and even live-animal disease models. Organoids arise from organ-derived adult pluripotent stem cells, organ stem cells, or cancer stem cells which possess the innate capacity to expand and differentiate into multiple cell types. Organoids generated from dozens of tissues and organs available commercially, or accessible through published protocols include patient-derived models of liver, heart, pancreas, brain, GI tract, kidney, and recently, of human airwayssuitable for drug and vaccine development and for studying infectious human respiratory diseases.

Corning Life Sciences has collaborated with HUB since 2014 to provide advanced organoids and related technology.

Dr Clevers technology allowed, for the first time, the expansion of adult stem cell-derived organoids in genetically stable form and ultimately, the generation of in vitro models of any epithelial disease from any patient.

A second key benefit was indefinite expansion similar to that of transformed cells, but without the genetic abnormalities inherent in cancer cells. Previously, organoids were generated from embryonic or induced pluripotent stem cells, or from tumor cells which by necessity are genetically modified and therefore unrepresentative of the patient.

Under HUBs commercial development, organoid technology also provides standardization and consistency which is difficult to match, especially with primary cell cultures. Biopsies from the same patient collect differing quantities of cells at widely varying stages of cell lifecycle. When cultured under identical HUB protocols adult progenitor cells give rise to organoids with exactly the same cells in the same proportions, physical configuration, and genetics, every time, and with broad expansion capabilities.

Similarly, transformed cells grown on plastic have modified their gene expression to adapt to tissue culture conditions. Studies with such cells can be useful, provided investigators recognize that the patients original genetics have not been preserved. In HUB organoids the patients molecular footprint is maintained.

One field where this has been particularly useful is infectious diseases. Viruses have evolved to infect and replicate in cells in their normal physiological states. For example, respiratory syncytial virus (RSV) readily grows in organoids but will not infect transformed cells because the cells lack the relevant receptors.

Cell-based studies of airway diseases topical in light of the current COVID-19 pandemic were hampered for years for this reason, and technology for expanding primary cell cultures sufficiently for large-scale studies did not exist. By preserving critical cell surface receptors for infectious agents, the HUB method allows the study of such pathogens as RSV, human papillomavirus, norovirus, coronavirus, influenza, malaria and many others.

Epithelial cells are the first point of contact for pathogenic microbes in the respiratory tract, and fortuitously the cell types most easily grown as organoids. Receptors on airway epithelia and alveolar cells sense infection, which initiates mucosal barrier immunity through club, ciliated, basal, goblet and neuroendocrine cells, which together clear inhaled pathogens.

In a recent Science paper, researchers from the Hubrecht Institute and Erasmus Medical Center reported on how gut organoids helped them to uncover two potential avenues for treating or preventing infection with SARS-CoV-2, the coronavirus responsible for the current pandemic. SARS-CoV-2 is known to infect the lungs, but clinical evidence suggests intestinal involvement in both symptomatology and transmission. For example, rectal swabs contain viral RNA for a time after nasal swabs indicate the infection has resolved, suggesting gastro-intestinal infection and possibly fecaloral transmission.

Differentiated enterocytes strongly express the SARS-CoV-2 angiotensin converting enzyme 2 (ACE2) receptor through which the virus enters cells, with the highest receptor levels found in the brush border of intestinal enterocytes. Surprisingly, virus infected both high- and low expressors of ACE2, and infectivity of organoids was not greatly affected by culture conditions.

SARS-CoV-2 rapidly infected a subset of cells within the organoid, and infection increased over time. Using electron microscopy to visualize cellular components, the researchers found virus particles inside and outside the organoids constituent cells. Infection induced release of interferon, an endogenous antiviral whose activation could serve as the target for potential therapies.

The researchers concluded that intestinal epithelium supports SARS-CoV-2 replication, that human small intestinal organoids serve as an experimental model for coronavirus infection and biology, and that human organoids represent faithful experimental models to study the biology of coronaviruses.

In addition to drug screening and toxicology studies, airway organoids have been utilized to study the basic biology of infectious diseases. In an application note, Corning scientists reported that Corning Matrigel extracellular matrix facilitated the expansion of patient-derived bronchial epithelial cells into airway organoids suitable for high throughput analysis. Organoids streamlined the usual sample preparation protocol to a single operation cell lysis eliminating the normal steps of gene amplification, cDNA conversion, and library preparation.

Comparing normal and asthmatic airway organoids, investigators observed increased expression of genes coding for pro-inflammatory chemokines, receptors, and other proteins associated with inflammation in asthmatic airway cells. They also found that the genes upregulated in organoids derived from healthy cells were the same as those downregulated in organoids from asthmatic cells, and vice-versa. Application of the anti-inflammatory steroid dexamethasone induced up- or downregulation to a greater degree in asthmatic organoids compared with normal organoids.

The Corning study illustrates the versatility of organoids for studying airway diseases in the presence of comorbidities, as well as the ability to respond rapidly with suitable models for infectious diseases.

HUB Organoids derived from adult stem cells harvested from cystic fibrosis patients have proved valuable in the study of CF pathology, and have permitted patient-centered drug testing, which was the first use of HUB Organoids in personalized medicine. The CF patient derived organoids are tested to identify drug treatments for CF patients and in treated accordingly.

Recent studies on interleukin-17 receptors on lung epithelia have uncovered a role for this cytokine in acute and chronic inflammation, and demonstrated that IL-17 receptors participate in the innate immune defense against pulmonary fungal infections. In vivo, IL-17 expression and immune function requires polarized epithelial cells. In a paper appearing in 2019 in Frontiers in Immunology, a group at the University of Perugia, in Italy, wrote that because lung organoids recapitulate tissue polarity, they provide an exciting possibility of using lung organoids to comprehensively investigate IL-17R signaling in the lung, which is likely to offer new opportunities to develop and test therapeutics for inflammatory diseases and identify new molecular targets to improve resistance to infections.

As a scientific discipline, organoids will continue evolving towards greater ease of use, consistency, assay parallelism capabilities, and manufacturability. Organoids and organ-on-a-chip have already been combined in a complex, multi-tissue retina model, while systems consisting of organoids from two or more organs, discussed earlier, are already used routinely.

If organoid research continues at its current pace there is reason to expect significant streamlining of early-stage drug development, specifically around the preclinical and phase 1 stages. Organoids could eliminate some if not all animal testing, but this will require a leap of faith on the part of regulators already accustomed to reviewing animal data and its inherent caveats. At some point organoids might completely eliminate live preclinical screens, allowing drug developers to recruit patients directly into phase 2 based entirely on organoid-based screening.

While organoid investigations inevitably lead to systems of greater complexity, investigators should keep in mind that validation is the key to patient relevant models. HUB Organoids for the first time allow researchers to develop a model and directly test if and how it resembles the patient from which the tissue originated. With increasing complexity, the validation step should remain a focus of model developers and users. Complexity is good, but only up to a point.

Advancing organoids towards these lofty goals, including greater manufacturability, will require cell culture tools up to the task. Industry collaborations assure that tools for 3D cell culture will continue to advance, both for general research and to meet the challenges of emerging infectious diseases.

Authors: Dr Robert Vries, Chief Executive Officer, Hubrecht Organoid Technology (HUB) Elizabeth Abraham, Senior Product Manager, Corning Incorporated

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Using Organoids in the Study of Infectious Diseases - Technology Networks

Microengineered 3D pulmonary interstitial mimetics highlight a critical role for matrix degradation in myofibroblast differentiation – Science…

INTRODUCTION

Fibrosis is implicated in nearly 45% of all deaths in the developed world and plays a role in numerous pathologies, including pulmonary fibrosis, cardiac disease, atherosclerosis, and cancer (1). In particular, interstitial lung diseases, such as idiopathic pulmonary fibrosis (IPF), are fatal and incurable with a median survival of only 2 to 5 years (2). Often described as dysregulated or incessant wound healing, fibrosis involves persistent cycles of tissue injury and deposition of extracellular matrix (ECM) by myofibroblasts (MFs). These critical cellular mediators of fibrogenesis are primarily derived from tissue-resident fibroblasts (1). MFs drive eventual organ failure through excessive fibrous ECM deposition, force generation and tissue contraction, and eventual disruption of parenchymal tissue function (1). As organ transplantation remains the only curative option for late-stage disease, effective antifibrotic therapeutics that slow MF expansion or even reverse fibrosed tissue remain a major unmet clinical need. Undoubtedly, the limited efficacy of antifibrotic drugs at present underscores limitations of existing models for identifying therapeutics, the complexity of the disease, and an incomplete understanding of MF biology.

A strong correlation between lung tissue stiffening and worse patient outcomes suggests an important role for matrix mechanosensing in fibrotic disease progression (3). Preclinical models of fibrosis in mice have supported the link between tissue stiffening and disease progression. However, a precise understanding of how physical cues from the microenvironment influence MF differentiation in vivo is confounded by concurrent structural (e.g., collagen density and laminin/elastin degradation) and biochemical (e.g., matrix composition and inflammatory) changes to the microenvironment (4). Consequently, natural and synthetic in vitro tissue models have provided great utility for the study of MF mechanobiology. Seminal studies using natural type I collagen gels have elucidated the role of profibrotic soluble cues [e.g., transforming growth factor1 (TGF-1)] in promoting cell contractility, ECM compaction, and MF differentiation, and more recently, precision-cut lung slices, have emerged as a powerful tool to study the complexity of the pulmonary microenvironment in IPF (4, 5). However, their utility in identifying physical microenvironmental determinants of MF differentiation suffers from an intrinsic coupling of multiple biochemical and mechanical material properties (6). Rapid degradation kinetics (1 to 3 days) and resulting issues with material stability (1 to 2 weeks) further impede the use of natural materials for studying fibrogenic events and drug responses, which occur over weeks to months in in vivo models or years in patients (7, 8).

Synthetic hydrogels that are more resistant to cell-mediated degradation have provided a better controlled setting for long-term studies of disease-related processes (9). For example, synthetic hydrogel-based cell culture substrates with tunable stiffness have helped establish a paradigm for mechanosensing during MF differentiation in two-dimensions (2D), where compliant matrices maintain fibroblast quiescence in contrast to stiffer matrices that promote MF differentiation (10, 11). Extensive findings in 2D suggest a causal role for matrix mechanics (e.g., stiffness) during MF differentiation in vitro and potentially in human disease, but these models lack the 3D nature of interstitial spaces where fibrosis originates (12). The interstitium surrounding alveoli is structurally composed of two key components: networks of fibrous ECM proteins (namely, type I collagen fibers) and interpenetrating ground substance, an amorphous hydrogel network rich in glycosaminoglycans such as heparan sulfate proteoglycan. Mechanical cues from fibrotic ECM that promote MF differentiation may arise from changes to the collagen fiber architecture or the gel-like ground substance; whether matrix stiffness is a prerequisite for MF differentiation in 3D fibrous interstitial spaces remains unclear (13). Furthermore, the limited efficacy of antifibrotic therapies identified in preclinical and in vitro models of IPF motivates the development of 3D tissue-engineered systems with improved structural and mechanical biomimicry, relevant pharmacokinetics, and the potential to incorporate patient cells (9). Furthermore, recapitulating key features of the fibrotic progression in an in vitro setting that better approximates interstitial tissues could (i) improve our current understanding of MF mechanobiology and (ii) serve as a more suitable test bed for potential antifibrotic therapeutics.

Accordingly, here, we describe a microengineered pulmonary interstitial matrix that recapitulates mechanical and structural features of fibrotic tissue as well as key biological events observed during IPF progression. Design parameters of these engineered microenvironments were informed by mechanical and structural characterization of fibrotic lung tissue from a bleomycin mouse model. We then investigated the influence of dimensionality, matrix cross-linking/stiffness, and fiber density on TGF-1induced MF differentiation in our pulmonary interstitial matrices. Increased hydrogel cross-linking/stiffness substantially hindered MF differentiation in 3D in contrast to findings in 2D, while fibrotic matrix architecture (i.e., high fiber density) potently promoted fibroblast proliferation and differentiation into MFs. Long-term (21 days) culture of hydrogels with a fibrotic architecture engendered tissue stiffening, collagen deposition, and secretion of profibrotic cytokines, implicating fiber density as a potent fibrogenic cue in 3D microenvironments. Pharmacologic screening in fibrotic pulmonary interstitial matrices revealed matrix metalloproteinase (MMP) activity and hydrogel remodeling as a key step during 3D fibrogenesis, but not in traditional 2D settings. To explore the clinical relevance of our findings, we leveraged a multistep bioinformatics analysis of transcriptional profiles from 231 patients, highlighting increased MMP gene expression and enriched signaling domains associated with matrix degradation in patients with IPF. Together, these results highlight the utility of studying fibrogenesis in a physiologically relevant 3D hydrogel model, underscore the requirement of matrix remodeling in IPF, and establish a new platform for screening antifibrotic therapies.

To inform key design criteria for our pulmonary interstitial matrices, we began by characterizing mechanical properties of fibrotic interstitial tissue in a bleomycin-induced lung injury model in mouse. Nave C57BL/6 mice were intratracheally challenged with bleomycin to induce lung injury and subsequent fibro-proliferative repair, with saline-treated animals maintained as a control group. After 2 weeks, animals were sacrificed and lung tissue was dissected out, sectioned and stained, and then mechanically tested by atomic force microscopy (AFM) nanoindentation to map the stiffness of interstitial tissue surrounding alveoli. While single-dose bleomycin administration does not recapitulate human IPF, the fibro-proliferative response is well characterized and leads to MF differentiation, collagen deposition, and lung stiffening events that are reminiscent of what occurs in human disease over longer time scales. As previously documented (14), bleomycin treatment corresponded to an increase in the thickness of interstitial tissue regions surrounding alveoli, a structural change that occurred alongside matrix stiffening (Fig. 1, A and B); bleomycin-treated lungs had elastic moduli nearly fivefold greater than healthy control tissues. To generate synthetic hydrogels with elastic moduli tunable over this range, we functionalized a biocompatible and protein-resistant polysaccharide, dextran, with pendant vinyl sulfone groups amenable to peptide conjugation (termed DexVS; Fig. 1C). To permit cell-mediated proteolytic hydrogel degradation and thus spreading of encapsulated cells, we cross-linked DexVS with a bifunctional peptide (GCVPMSMRGGCG, abbreviated VPMS) primarily sensitive to MMP9 and MMP14, two MMPs implicated in fibrosis-associated matrix remodeling (15, 16). Tuning input VPMS cross-linker concentration yielded stable hydrogels spanning the full range of elastic moduli we measured by AFM nanoindentation of lung tissue (Fig. 1D). Additional functionalization with cell-adhesive moieties (CGRGDS, abbreviated RGD) facilitated adhesion of primary normal human lung fibroblasts (NHLFs) (Fig. 1E).

(A) Histological preparations of healthy control and bleomycin-treated murine lung tissue (n = 3 mice per group) stained for collagen by picrosirius red (scale bar, 100 m). (B) Youngs modulus of mouse lung tissue as measured by AFM nanoindentation, with data fit to the Hertz contact model to determine Youngs modulus (n = 3 mice per group, n = 50 indentations per group on n = 9 tissue sections). (C) Schematic of proteolytically sensitive, cell-adhesive DexVS-VPMS bulk hydrogels. (D) Youngs modulus determined by AFM nanoindentation of DexVS-VPMS hydrogels formed with different concentrations of VPMS cross-linker (n = 4 samples per group, n = 20 total indentations per group). (E and F) Representative images of F-actin (cyan), nuclei (yellow), and -SMA (magenta); image-based quantification of -SMA expression (left axis, magenta bars, day 9) and nuclear Ki67 (right axis, gray bars, day 5) in 2D and 3D (n = 4 samples per group, n = 10 fields of view per group, n > 50 cells per field of view; scale bars, 200 m). All data presented are means SDs with superimposed data points; asterisk denotes significance with P < 0.05 determined by one-way analysis of variance (ANOVA). AU, arbitrary units.

To confirm the role of matrix mechanics on cell proliferation and MF differentiation, we seeded patient-derived NHLFs on 2D DexVS protease-sensitive hydrogel surfaces varying in VPMS cross-linker density and resulting stiffness and stimulated cultures with TGF-1 to promote MF differentiation. In accordance with previous literature, we observed a stiffness-dependent stepwise increase in cell proliferation (day 5) and MF differentiation (day 9) as measured by Ki67 and -smooth muscle actin (-SMA) immunofluorescence, respectively (Fig. 1E) (11). As the influence of matrix elasticity on MF differentiation in 3D synthetic matrices has not previously been documented, we also encapsulated NHLFs in 3D within identical DexVS hydrogels. The opposing trend with respect to stiffness was noted for cells encapsulated in 3D; compliant (E = 560 Pa) hydrogels that limited -SMA expression in 2D plated cells instead exhibited the highest levels of MF differentiation in 3D (Fig. 1F). Decreasing proliferation and cell-cell contact formation as a function of increasing hydrogel stiffness were also noted in 3D matrices and may be one reason why rigid hydrogels limit differentiation in 3D. Similar findings have been reported for mesenchymal stem cells encapsulated in hyaluronic acid matrices, where compliant gels promoted stem cell proliferation and yes-associated protein (YAP) activity in 3D, yet inhibited YAP activity and proliferation in 2D (17). These results suggest that while stiff, cross-linked 2D surfaces promote cell spreading, proliferation, and MF differentiation, an equivalent relationship does not directly translate to 3D settings. High cross-linking and stiffness (E = 6.1 kPa) in 3D matrices sterically hinder cell spreading, proliferation, and the formation of cell-cell contacts, all well-established promoters of MF differentiation (18).

Cell-degradable synthetic hydrogels with elastic moduli approximating that of fibrotic tissue proved nonpermissive to MF differentiation in 3D. Although matrix cross-linking and densification of ground substance has previously been implicated in fibrotic tissue stiffening, remodeled collagenous architecture can also engender changes in tissue mechanics and may modulate MF development in IPF independently. To characterize the fibrous matrix architecture within healthy and fibrotic lung interstitium, we used second-harmonic generation (SHG) microscopy to visualize collagen microstructure in saline- and bleomycin-treated lungs, respectively. Per previous literature, saline-treated lungs contained limited numbers of micrometer-scale (~1-m-diameter) collagen fibers, primarily localized to the interstitial spaces supporting the alveoli (Fig. 2A) (19). In contrast, bleomycin-treated lungs had, on average, fourfold higher overall SHG intensity, with collagen fibers localized to both an expanded interstitial region and in disrupted alveolar networks. While no difference in fiber diameter was noted with bleomycin treatment, we did observe thick (~2- to 5-m) collagen bundles containing numerous individual fibers in fibrotic lungs, potentially arising from physical remodeling by resident fibroblasts (Fig. 2A and fig. S1). Given that typical synthetic hydrogels amenable to cell encapsulation (as in Fig. 1) lack fibrous architecture, we leveraged a previously established methodology for generating fiber-reinforced hydrogel composites (20). Electrospun DexVS fibers approximating the diameter of collagen fibers characterized by SHG imaging (fig. S1) were co-encapsulated alongside NHLFs in DexVS-VPMS hydrogel matrices, yielding a 3D interpenetrating network of DexVS fibers ensconced within proteolytically cleavable DexVS hydrogel (Fig. 2B). To recapitulate the adhesive nature of collagen and fibronectin fibers within interstitial tissues, we functionalized DexVS fibers with RGD to support integrin engagement and 3D cell spreading. While increasing the weight % of type I collagen matrices increases collagen fiber density and simultaneously increases hydrogel stiffness (fig. S2), our synthetic matrix platform enables changes to fiber density (0.0 to 5.0%) without altering mechanical properties assessed by AFM nanoindentation (Fig. 2C), likely due to the constant weight percentage of DexVS and VPMS cross-linker within the bulk hydrogel.

(A) SHG imaging of collagen microstructure within healthy and bleomycin-treated lungs on day 14, with quantification of average signal intensity (arrows indicate interstitial tissue regions adjacent to alveoli; n = 3 mice per group, n = 10 fields of view per group; scale bar, 100 m). (B) Schematic depicting polymer cross-linking and functionalization for generating fibrous DexVS hydrogel composites to model changes in fiber density within lung interstitial tissue ECM. (C) Images and intensity quantification of fluorophore-labeled fibers within composites varying in fiber density (n = 4 samples per group, n = 10 fields of view per group; scale bar, 100 m). Youngs modulus determined by AFM nanoindentation of fibrous composites formed with different concentrations of VPMS cross-linker (n = 4 samples per group, n = 20 measurements per group). (D) Representative high-resolution images of NHLFs on day 1 in fibrous composites formed with bulk hydrogels (12.5 mM VPMS) functionalized with integrin ligand arginylglycylaspartic acid (RGD) or heparin-binding peptide (HBP) [F-actin (cyan), nuclei (yellow), and DexVS fibers (magenta); scale bar, 50 m]. Quantification of fiber recruitment as measured by contact between cells and DexVS fibers (n = 10 fields of view per group, n > 25 cells analyzed). (E) Representative high-resolution images of NHLF on day 1 fibrous composites formed with bulk hydrogels functionalized with integrin ligand RGD or HBP [F-actin (cyan), fibronectin (yellow), and DexVS fibers (magenta); scale bar, 5 m]. Quantification of fibronectin deposition into tshe hydrogel matrix as measured by immunostain intensity (n = 10 fields of view per group, n > 25 cells analyzed). All data presented are means SDs with superimposed data points; asterisk denotes significance with P < 0.05 determined by one-way ANOVA or Students t test, where appropriate; NS denotes nonsignificant comparison.

Beyond recapitulating the multiphase structural composition of interstitial ECM, we also sought to mimic the adhesive ligand presentation and protein sequestration functions of native interstitial tissue. More specifically, the gel-like ground substance within fibrotic tissue intrinsically lacks integrin-binding moieties and is increasingly rich in heparan sulfate proteoglycans, primarily serving as a local reservoir for nascent ECM proteins, growth factors, and profibrotic cytokines. In contrast, synthetic hydrogels are often intentionally designed to have minimal interactions with secreted proteins and require uniform functionalization with a cell-adhesive ligand to support cell attachment and mechanosensing. We hypothesized that RGD-presenting fibers alone would support cell spreading (20), enabling the use of a nonadhesive bulk DexVS hydrogel functionalized with heparin-binding peptide (HBP; CGFAKLAARLYRKAG) (21). While both RGD- and HBP-functionalized bulk DexVS gels supported cell spreading upon incorporation of RGD-presenting fibers, HBP-functionalized hydrogels encouraged matrix remodeling in the form of cell-mediated fiber recruitment (Fig. 2D) and enhanced the deposition of fibronectin fibrils into the adjacent matrix (Fig. 2E). Given the multiphase structure of lung interstitium, changes in collagen fiber density noted with fibrotic progression, and the importance of physical and biochemical matrix remodeling to fibrogenesis, we used HBP-tethered 560-Pa DexVS-VPMS bulk hydrogels with tunable density of RGD-presenting fibers in all subsequent studies.

We next investigated whether changes in fiber density reflecting fibrosis-associated alterations to matrix architecture could influence MF differentiation in our 3D model. NHLFs were encapsulated in compliant DexVS-VPMS hydrogels ranging in fiber density (E = 560 Pa, 0.0 to 5.0 volume % fibers). Examining cell morphology after 3 days of culture, we noted increased cell spreading (Fig. 3, A and B) and evident F-actin stress fibers (fig. S3) in fibrous conditions compared to nonfibrous controls. Increased frequency of direct cell-cell interactions was also observed as a function of fiber density, as evidenced by higher area:perimeter ratios and the number of fibroblasts per contiguous multicellular cluster (Fig. 3A and fig. S3). As evidenced by changes in the ratio of nuclear to cytosolic YAP localization, we detected changes in mechanosensing as a function of fiber density, with the highest nuclear ratio measured in samples containing the highest fiber density examined. Given that nuclear YAP activity (a transcriptional coactivator required for downstream mechanotransduction) has been implicated as a promoter of MF differentiation (22), we also assayed other markers associated with fibroblast activation. With increases in fiber density, we found significant increases in cell proliferation and local fibronectin deposition (Fig. 3, A and B). Luminex quantification of cytokine secretion at this time point revealed elevated secretion of inflammatory and profibrotic cytokines (Fig. 3C), suggesting that matrix fibers may modulate the soluble milieu known to regulate the response to tissue damage and repair in vivo (2325). While no -SMA expression or collagen deposition was observed at this early time point, F-actin stress fibers, YAP activity, and fibronectin expression have been previously established as proto-MF markers in vivo (26), suggesting that physical interactions with matrix fibers prime fibroblasts for activation into MFs. Supplying the profibrotic soluble factor TGF-1 prompted increases in the expression of various profibrotic YAP-target genes (ACTA2, COL1A1, FN1, CD11, and CTGF) relative to nonfibrous (FD 0.0%) controls at day 5 (Fig. 3D). Together, these data suggest that heightened fiber density promotes a fibrotic phenotype (Fig. 3, A to C) and gene expression (Fig. 3D), despite the absence of a stiff surrounding hydrogel.

(A) Immunofluorescence images of NHLFs in hydrogel composites over a range of fiber densities after 3 days of culture [F-actin (cyan), fibronectin (FN, yellow), YAP (magenta), Ki67 (white), and nuclei (blue); scale bars, 100 m (F-actin), 20 m (FN), 20 m (YAP), and 100 m (Ki67/nuclei)]. (B) Corresponding image-based quantification of cell area, deposited FN, YAP nuclear to cytosolic ratio, and % of proliferating cells (n = 4 samples per group; for cell spread area analysis, n > 50 cells per group; for FN, YAP, and Ki67 analyses, n = 10 fields of view per group and n > 25 cells per field of view). (C) Cytokine secretion into culture medium on day 3 (all data were normalized to background levels in control medium, n = 4 samples per condition). (D) Expression of MF-related genes in NHLFs stimulated with TGF-1 on day 3, in either highly fibrous (FD 5.0%) or nonfibrous (FD 0.0%) hydrogels (data presented are GAPDH-normalized fold changes relative to NHLFs within an FD 0% hydrogel lacking TGF-1 supplementation). All data presented are means SDs with superimposed data points; asterisk denotes significance with P < 0.05 determined by one-way ANOVA or Students t test where appropriate.

To explore whether fibrotic matrix cues in the form of heightened fiber density could promote 3D MF differentiation over longer-term culture, NHLFs were encapsulated within hydrogels varying in fiber density and maintained in medium supplemented with TGF-1 beginning on day 1. Immunofluorescent imaging and cytokine quantification were performed on days 3, 5, 7, and 9 to capture dynamic changes in cellular phenotype and secretion, respectively. No -SMApositive stress fibers or changes in total cytokine secretion were observed on day 3 or 5. On day 7, we noted the sparse appearance of -SMApositive cells alongside increased total cytokine secretion (Fig. 4D) in FD 5.0% conditions containing TGF-1, indicating the beginning of a potential phenotypic shift. Extensive MF differentiation (designated by -SMApositive cells) and a sixfold increase in total cytokine secretion occurred rapidly between days 7 and 9 (Fig. 4, B, D, and E) in the highest fiber density (FD 5.0%) condition. Despite the high proliferation within high fiber density hydrogels (Fig. 4C), -SMApositive cells were not evident in samples lacking exogenous TGF-1 supplementation. Moreover, -SMApositive cells were also absent in TGF-1 supplemented conditions that lacked fibrous architecture, indicating a requirement for both soluble and physical fibrogenic cues in 3D. Furthermore, inhibiting integrin engagement by incorporating fibers lacking RGD also abrogated MF differentiation and proliferation despite the presence of TGF-1 (Fig. 4, A and B), suggesting that a fibrotic matrix architecture drives -SMA expression primarily through integrin engagement and downstream mechanosensing pathways. These results were replicated with primary human dermal fibroblasts and mammary fibroblasts, where similar trends with -SMA expression as a function of fiber density were observed (fig. S4). While high fiber density promoted proliferation in dermal fibroblasts, mammary fibroblasts underwent MF differentiation in the absence of higher proliferation rates, demonstrating intrinsic differences between cell populations originating from different tissues. Nevertheless, these results suggest that fibrotic matrix architecture may be promoting MF differentiation in other pathologies, namely, dermal scarring in systemic sclerosis and desmoplasia in breast cancer.

(A) Representative immunofluorescence images of NHLFs in microenvironmental conditions leading to low (top row) or high (bottom row) MF differentiation after 9 days in culture [-SMA (magenta) and nuclei (cyan); n = 4 samples per group, n = 10 fields of view per group, and n > 50 cells per field of view; scale bar, 200 m], with corresponding image-based quantification in (B) and (C). Insets depict representative fiber densities. (D) Measurement of total cytokine secretion over time as a function of fiber density (n = 4 samples per condition; * indicates significant differences between FD 5.0% and all other groups at a given time point; NS denotes nonsignificant comparison). (E) Secretion of specific cytokines and chemoattractants as a function of fiber density on day 9 (n = 4 samples per condition). (F) Representative images and quantification of tissue contraction within day 14 fibroblast-laden hydrogels of varying fiber density (n = 4 samples per group, dashed line indicates initial diameter of 5 mm). Photo credit: Daniel Matera, University of Michigan. (G) AFM measurements of day 14 fibroblast-laden hydrogels of varying fiber density (n = 20 measurements from n = 4 samples per group). Dashed line indicates original hydrogel stiffness. (H) SHG images of fibrous collagen within fibroblast-laden hydrogels after 21 days of culture in medium supplemented with ascorbic acid (scale bar, 100 m). (I) Measurement of total collagen content within digested DexVS hydrogels at day 21 as measured by biochemical assay (n = 4 samples per group). All data presented are means SDs with superimposed data points; asterisk denotes significance with P < 0.05 determined by one-way ANOVA; NS denotes nonsignificant comparison.

While proliferation and -SMA expression are accepted markers of activated fibroblasts, fibrotic lesions contribute to patient mortality through airway inflammation, collagen secretion, tissue contraction, and lung stiffeningpathogenic events that hinder the physical process of respiration (27). Luminex screening of 41 cytokines and chemokines within hydrogel supernatant revealed elevated total cytokine secretion as a function of fiber density over time (Fig. 4D), many of which were soluble mediators known to regulate airway inflammation (Fig. 4E) (23). Numerous other cytokines were additionally secreted at day 9 but did not change as a function of fiber density despite differences in cell number at this time point (fig. S5), suggesting that cell number alone cannot account for the increased cytokine secretion in high fiber density conditions. By generating free-floating hydrogels that allow contraction over time, we also examined macroscale changes in tissue geometry. Consistent with the influence of fiber density on -SMA expression, hydrogels containing high fiber densities underwent greater hydrogel contraction compared to nonfibrous or low fiber density conditions (Fig. 4F). Day 14 fibrotic hydrogels (FD 5.0%) were also fourfold stiffer (2.0 versus 0.5 kPa) as measured by AFM nanoindentation (Fig. 4G) compared to conditions that yielded low rates of MF differentiation in shorter-term studies (i.e., FD 0.0 or FD 0.5% in Fig. 4, A and B). When medium was supplemented with ascorbic acid to permit procollagen hydroxylation, collagen deposition into the surrounding matrix was evident by SHG microscopy by day 21 in high fiber density hydrogels (Fig. 4H) as compared to nonfibrous controls. Further biochemical analysis of hydrogel collagen content confirmed a stepwise increase in collagen production as a function of fiber density (Fig. 4I). Together, these findings demonstrate a clear influence of fiber density on MF differentiation and phenotype in 3D and furthermore suggest that this in vitro model recapitulates key pathogenic events associated with the progression of fibrosis in vivo.

Having established microenvironmental cues that promote robust 3D MF differentiation, we next evaluated the potential of our fibrous hydrogel model for use as an antifibrotic drug screening platform. Nintedanib, a broad-spectrum receptor tyrosine kinase inhibitor, and pirfenidone, an inhibitor of the mitogen-activated protein kinase (MAPK)/nuclear factor B (NF-B) pathway, were selected due to their recent Food and Drug Administration approval for use in patients with IPF (28). We also included dimethyl fumarate, an inhibitor of the YAP/TAZ pathway clinically approved for treatment of systemic sclerosis, and marimastat, a broad-spectrum MMP inhibitor that has shown efficacy in murine preclinical models of fibrosis (29, 30). We generated fibrotic matrices (560-Pa DexVS-VPMS-HBP bulk hydrogels containing 5.0 volume % DexVS-RGD fibers) that elicited the highest levels of MF differentiation, matrix contraction, and collagen secretion in our previous studies (Fig. 4). As a comparison to the current standard for high-throughput compound screening, we also seeded identical numbers of NHLFs on 2D tissue culture plastic in parallel. Cultures were stimulated with TGF-1 on day 1, and pharmacologic treatments were added on day 3, following extensive fibroblast spreading, cell-cell junction formation, and proliferation (Fig. 3A).

As in our earlier studies, TGF-1 supplementation promoted proliferation and -SMA expression within 3D constructs as well as on rigid tissue culture plastic (Fig. 5A). Nintedanib and pirfenidone had differential effects on NHLFs depending on culture format; NHLFs on 2D tissue culture plastic were resistant to pirfenidone/nintedanib treatment with no difference in proliferation or -SMA expression relative to vehicle controls, whereas modest but significant decreases in -SMA expression (pirfenidone and nintedanib) and proliferation (nintedanib) were detected in 3D (Fig. 5, A to E). Combined treatment with pirfenidone and nintedanib provided an antifibrotic effect only in fibrotic matrices, supporting ongoing clinical studies exploring their use as a combinatorial therapy (ClinicalTrials.gov identifier NCT03939520). Dimethyl fumarate abrogated cell proliferation and -SMA expression across all conditions, suggesting that inhibition of downstream mechanosensing inhibits MF differentiation in both 2D and 3D contexts in support of the general requirement for mechanosensing during MF differentiation independent of culture substrate (11). Inhibition of YAP activity in vivo has been shown to mitigate fibrosis and may be an advantageous therapeutic target (22). Blockade of MMP activity via marimastat treatment proved ineffectual in reducing -SMA expression or proliferation on 2D tissue culture plastic, but surprisingly fully abrogated the proliferation and differentiation response in 3D fibrotic matrices (Fig. 5, A to E). Given the role of protease activity in tissue remodeling in vivo (30) and in cellular outgrowth within 3D hydrogels (17, 31), our data suggest that degradative matrix remodeling is a requirement for MF differentiation in 3D, but not in more simplified 2D settings. To summarize, multiple antifibrotic agents (pirfenidone, nintedanib, dimethyl fumarate, and marimastat) demonstrating efficacy in clinical literature elicited an antifibrotic effect in our engineered fibrotic pulmonary interstitial matrices, but not in the 2D tissue culture plastic contexts traditionally used for compound screening.

(A) Representative confocal images stained for -SMA (magenta), F-actin (cyan), and nuclei (yellow) of NHLFs after 9 days of culture on tissue culture plastic (TCP) (top row) or 3D fibrotic matrices (bottom row) with pharmacologic treatment indicated from days 3 to 9 (scale bar, 100 m). Imaged regions were selected to maximize the number of -SMA+ cells per field of view within each sample. (B) Quantification of -SMA and (C) total cell count within 2D NHLF cultures. (D) Quantification of -SMA and (E) total cell count within 3D fibrotic matrices (n = 4 samples per group, n = 10 fields of view per group, and n > 50 cells per field of view). All data presented are means SDs with superimposed data points; asterisk denotes significance with P < 0.05 determined by one-way ANOVA; NS denotes nonsignificant comparison.

As the protease inhibitor marimastat fully ablated TGF-1induced -SMA expression and proliferation in our 3D fibrotic matrices, we leveraged bioinformatics methodologies to investigate the role of matrix proteases in patients with IPF on a network (Reactome) and protein (STRING) basis. Differential expression analysis of microarray data within the National Center for Biotechnology Information (NCBI) Gene Expression Omnibus (GEO) (dataset #GSE47460) was used to generate an uncurated/unbiased dataset composed of the top 1000 differentially regulated genes in IPF, revealing MMP1 as the most up-regulated gene in patients with IPF, with other matrix proteases (MMP1, MMP3, MMP7, MMP9, MMP10, MMP11, and MMP12) and matrix remodeling proteins (COL1A2, LOX, ACAN, DCN, and HS6ST2) similarly up-regulated (Fig. 6B, table S1, and data file S1). To focus on genes associated with MF differentiation for subsequent analyses, we performed Gene Ontology (GO) term enrichment (via GEO2R) to compile a curated dataset containing 188 key genes associated with MF differentiation (data file S1) and used Reactome and STRING analyses to investigate network signaling within both the uncurated and curated datasets. Analyses revealed 103 (uncurated) and 89 (curated) enriched signaling pathways in IPF (data file S1). The top 3/5 (uncurated) and 5/5 (curated) significantly enriched pathways in IPF involved matrix degradation and remodeling (Fig. 6C). Subsequent STRING protein-protein interaction analysis of datasets revealed that top signaling nodes were MMPs (uncurated: MMP1 and MMP3; Fig. 6D), fibrous collagens (uncurated: COL1A2 and COL3A1), or cytokines (curated: IL6, VEGFA, IL1B, and IGF1; Fig. 6D) known to increase MMP expression in fibroblasts (3235). These results emphasize the interdependence between MMP activity and systems-level pathogenic signaling in IPF and, in combination with our 3D drug screening results, highlight fibroblast-specific protease activity as a potential therapeutic target. Furthermore, given that protease inhibition had no effect on MF differentiation in 2D culture, these data also support the growing sentiment that simplified 2D screening models may be masking the identification of potentially viable antifibrotics.

(A) Schematic representation of bioinformatics workflow: Whole-genome transcriptomes from 91 healthy and 140 patients with lung fibrosis were fetched from the NCBI GEO. Differential expression analysis was used to assemble an uncurated list of the top 1000 differentially expressed genes. GO enrichment of choice biological pathways was used to assemble a curated list of genes associated with MF differentiation. Datasets were fed through a previous knowledgebased analysis pipeline to identify enriched signaling pathways (Reactome) and key protein signaling nodes (STRING) within patients with IPF. (B) Heatmaps of the top 20 differentially expressed genes within specified GO categories, which were manually selected for curated analysis. CN values indicate a high degree of interaction between proteins selected for curated analysis. Colors are based on differential expression values that were not log-normalized. (C) Summary of the top 5 significantly enriched pathways in the curated and uncurated gene set. (D) Representative STRING diagram depicting protein interactions within the curated dataset, with summary of the top 5 signaling nodes in the uncurated and curated gene set. Blue nodes and edges represent interactions within the top 5 signaling nodes for the curated dataset.

Despite fibrosis widely contributing to mortality worldwide, inadequate understanding of fibrotic disease pathogenesis has limited the development of efficacious therapies (12). Preclinical studies in vivo, while indispensable, often fail to translate to clinical settings as evidenced by the failure of ~90% of drugs identified in animal studies (36). In addition, limitations in current technologies (e.g., the embryonic lethality of many genetic ECM knockouts and the limited resolution/imaging depth of intravital microscopy) have hindered the application of preclinical in vivo models for the study of cell-ECM interactions that underlie fibrogenesis (37). In contrast, existing in vitro models use patient-derived cells that are affordable, scalable, and amenable to microscopy, but often fail to recapitulate the complex 3D matrix structure of the interstitial tissue regions where fibrotic diseases such as IPF originate. We leveraged electrospinning and bio-orthogonal chemistries to engineer novel pulmonary interstitial matrices that are 3D and have fibrous architecture with biomimetic ligand presentation. In the presence of profibrotic soluble factors, these settings reproduce hallmarks of fibrosis at cellular and tissue levels (Figs. 2 to 4). Examining the influence of physical microenvironmental cues (cross-linking/stiffness and fiber density) on MF differentiation, we find that cross-linking/stiffness has opposing effects on MF differentiation in 2D versus 3D (Fig. 1) and that incorporation of a fibrous architecture in 3D is a prerequisite to MF differentiation (Fig. 4). Furthermore, supported by the importance of protease signaling in IPF (Fig. 6), we performed proof-of-concept pharmacologic screening within our 3D fibrotic matrices (Fig. 5) and highlighted enhanced biomimicry as compared to traditional 2D drug screening substrates where matrix remodeling appears to be dispensable for MF differentiation.

While tunable synthetic hydrogels have identified mechanosensing pathways critical to MF differentiation in 2D, these observations have yet to be translated to 3D fibrous settings relevant to the interstitial spaces where fibrosis originates. Given that late-stage IPF progresses in the absence of external tissue damage, current dogma implicates fibrotic matrix stiffness as the continual driver of MF differentiation in vivo (10, 11, 38). While we cannot disregard this hypothesis, our work elucidates a contrasting MMP-dependent mechanism at play in 3D, whereby a compliant, degradable, and fibrous matrix architecture supports MF differentiation, with matrix contraction and stiffening occurring downstream of -SMA expression, nearly a week later. Given numerous 2D studies indicating matrix stiffness as a driver of MF differentiation, the finding that a compliant matrix promotes MF differentiation may appear counterintuitive (10, 11). However, MF accumulation has been documented before tissue stiffening in human disease (3), and a recent phase 2 clinical trial (ClinicalTrials.gov Identifier: NCT01769196) targeting the LOX pathway (the family of enzymes responsible for matrix stiffening in vivo) failed to prevent disease progression in patients with IPF and was terminated due to lack of efficacy (39). Furthermore, compelling recent work by Fiore et al. (3) combined immunohistochemistry with high-resolution AFM to characterize human IPF tissue mechanics and found that regions of active fibrogenesis were highly fibrous but had a similar Youngs modulus as healthy tissue. In concert with our in vitro data, these findings suggest that MF differentiation is possible within soft provisional ECM in vivo and that the initiation of fibrogenesis may not be dependent on heightened tissue stiffness so long as matrix fibers and appropriate soluble cues (e.g., TGF-1) are present.

Consequently, understanding the source of profibrotic soluble cues in vivo is of critical importance when identifying therapeutic targets for IPF. Luminex screening of supernatant from 3D fibrotic matrices revealed sixfold increases in cytokine secretion during fibrogenesis, most of which were potent inflammatory factors [e.g., granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-6 (IL-6), IL-8, and vascular endothelial growth factor A (VEGF-A)] and chemoattractants (e.g., CCL2, CCL7, CCL11, and CXCL1) (Fig. 4E). Furthermore, IL-6 and VEGF-A were found to be major signaling nodes in patients with IPF (Fig. 6D). While not typically regarded as an immunomodulatory cell population, these findings suggest that MFs may maintain localized inflammation to support continual fibrogenesis. Mitogens such as IL-6 and IL-8 promote endothelial- and epithelial-to-mesenchymal transition, a process that gives rise to matrix-producing MF-like cells in IPF (40). CCR2 (CCL2 and CCL7) and CXCR1 (CXCL1 and IL-8) ligation facilitates macrophage chemotaxis, potentially leading to a sustained influx of TGF-1producing cells in IPF, and glycoproteins such as GM-CSF inhibit caspase activity in mononuclear cells, potentially preventing apoptotic events required for the resolution of wound repair and return to homeostasis (23, 41). In addition, secretion of nearly all cytokines was increased as a function of fiber density, highlighting a potential feed-forward loop distinct from canonical TGF-1 signaling. Further model development (e.g., coculture platforms) will be required to examine these hypotheses and the role of MF-derived cytokines in persistent inflammation and fibrosis.

In addition to documenting the role of fibrotic matrix architecture in 3D fibrogenesis, we demonstrate proof-of-concept pharmacologic screening within our synthetic pulmonary interstitial matrices and highlight their improved relevance to human disease. Previous work in vitro has documented profound reductions in MF differentiation after treatment with clinically approved antifibrotics (pirfenidone and nintedanib), whereas in the clinic, pirfenidone and nintedanib impede disease progression but are far from curative (4, 28, 42, 43). Pirfenidone or nintedanib had insignificant effects in 2D settings in our hands and only modest effects in 3D (Fig. 5). One reason for this discrepancy may be the use of supraphysiologic pirfenidone and nintedanib concentrations in previous in vitro studies, whereas we selected dosages based on plasma concentrations in patients with IPF (44). Differences in pharmacokinetics, nutrient/growth factor diffusion, and cell metabolism between 2D and 3D tissue constructs likely also play a role. Furthermore, as evidenced by the preventative effect of the protease inhibitor marimastat in 3D hydrogels but not 2D settings (Fig. 5), pharmacologics that influence matrix degradation and remodeling are likely to have a minimized effect in 2D settings due to the less dynamic nature of tissue culture plastic and flat hydrogels (45). Nintedanib and pirfenidone have been shown to influence protease activity and matrix remodeling in vivo (16), and may be mediating their effects within fibrotic matrices through modulation of ECM remodeling. Given the identification of numerous potential antifibrotic agents (microRNA, TGF-1 inhibitors, IL-4, IL-13 neutralizing antibodies, and integrin blockers) in preclinical models, application of the system described here could elucidate how choice pharmacologics affect MF differentiation and matrix remodeling processes that are difficult to recapitulate in 2D culture. Further development of our interstitial matrices as an arrayed platform, as has been elegantly implemented with collagen matrices (42), is a critical next step to moving this technology toward high-throughput screening applications.

It is important to note that this work has several potential limitations. Our material approach allows facile control of initial microenvironmental conditions (e.g., dimensionality, fiber density, ligand density, and elastic modulus), and of note, composites of RGD-bearing nondegradable fibers and degradable bulk hydrogel decouple degradation-induced changes in matrix mechanics and ligand availability. However, we have no experimental control over subsequent dynamic cell-driven remodeling events (e.g., MMP-mediated hydrogel softening, fibronectin and collagen deposition, and hydrogel contraction/stiffening from resident cells) that likely affect local matrix mechanics, cellular mechanosensing, and MF differentiation. Exciting recent technologies such as 3D traction force microscopy (TFM) and magnetic bead microrheology could enable future examination of how these dynamic changes in cell-scale mechanics potentiate MF differentiation in 3D. Along similar lines, although our study suggests a requirement for initial adhesion to the surrounding matrix, how the dynamics of ligand presentation due to matrix remodeling regulates mechanosensing was not explored here. We present this platform as a reductionist approach to modeling the activation of fibroblasts within the 3D fibrous interstitia associated with fibrosis, a pathology that develops over years in vivo and involves multiple cell types. Human pulmonary tissue and fibrotic foci, in particular, also have viscoelastic and nonlinear mechanical behaviors (3, 46) that were not explored in our AFM measurements of murine lung or hydrogel composites. Given the important role such mechanical features can play in ECM mechanosensing, incorporating new synthetic material strategies in combination with cell-scale mechanical measurements will be essential to modeling physiologic complexity. Given that the development of lung organoids is still in its infancy, decellularized precision-cut lung slices currently represent the best culture platform to capture the full complexity of the lung microenvironment (5).

In summary, we designed a tunable 3D and fibrous hydrogel model that recapitulates dynamic physical (e.g., stiffening and contraction) and biochemical (e.g., secretion of fibronectin, collagen, and cytokines) alterations to the microenvironment observed during the progression of IPF. Implementation of our model allowed us to establish a developing mechanism for MF differentiation in 3D compliant environments, whereby cell spreading upon matrix fibers drives YAP activity, cytokine release, and proteolysis-dependent MF differentiation. Furthermore, we leveraged bioinformatics techniques to explore protease signaling in clinical IPF and, in concert with our therapeutic screening data, establish a strong role for proteases during IPF pathogenesis and in 3D MF differentiation. Whether protease activity promoted MF differentiation directly through modulation of intracellular signaling or indirectly through affects on the local matrix environment has yet to be explored in these settings but will be the focus of future efforts. Consequently, these results highlight critical design parameters (3D degradability and matrix architecture) frequently overlooked in established synthetic models of MF differentiation. Future work incorporating macrophages, endothelial cells, and epithelial cells may expand current understanding of how developing MF populations influence otherwise homeostatic cells and how matrix remodeling influences paracrine signaling networks and corresponding drug response. Given the low translation rate of drugs identified in high-throughput screening assays, we show that the application and development of engineered biomimetics, in combination with preclinical models, can improve drug discovery and pathophysiological understanding.

All reagents were purchased from Sigma-Aldrich and used as received, unless otherwise stated.

Dextran vinyl sulfone. A previously described protocol for vinyl sulfonating polysaccharides was adapted for use with linear highmolecular weight (MW) dextran (MW 86,000 Da; MP Biomedicals, Santa Ana, CA) (20). Briefly, pure divinyl sulfone (12.5 ml; Thermo Fisher Scientific, Hampton, NH) was added to a sodium hydroxide solution (0.1 M, 250 ml) containing dextran (5 g). This reaction was carried out at 1500 rpm for 3.5 min, after which the reaction was terminated by adjusting the pH to 5.0 via the addition of hydrochloric acid. A lower functionalization of DexVS was used for hydrogels, where the volume of divinyl sulfone reagent was reduced to 3.875 ml. All reaction products were dialyzed for 5 days against Milli-Q ultrapure water, with two water exchanges daily, and then lyophilized for 3 days to obtain the pure product. Functionalization of DexVS was characterized by 1H nuclear magnetic resonance (NMR) spectroscopy in D2O and was calculated as the ratio of the proton integral [6.91 parts per million (ppm)] and the anomeric proton of the glucopyranosyl ring (5.166 and 4.923 ppm); here, vinyl sulfone/dextran repeat unit ratios of 0.376 and 0.156 were determined for electrospinning and hydrogel DexVS polymers, respectively.

DexVS was dissolved at 0.6 g ml1 in a 1:1 mixture of Milli-Q ultrapure water and dimethylformamide with 0.015% Irgacure 2959 photoinitiator. Methacrylated rhodamine (0.5 mM; Polysciences Inc., Warrington, PA) was incorporated into the electrospinning solution to fluorescently visualize fibers under 555 laser. This polymer solution was used for electrospinning within an environment-controlled glovebox held at 21C and 30% relative humidity. Electrospinning was performed at a flow rate of 0.3 ml hour1, gap distance of 5 cm, and voltage of 10.0 kV onto a grounded collecting surface attached to a linear actuator. Fiber layers were collected on glass slabs and primary cross-linked under ultraviolet light (100 mW cm2) and then secondary cross-linked (100 mW cm2) in an Irgacure 2959 solution (1 mg ml1). After polymerization, fiber segments were resuspended in a known volume of phosphate-buffered saline (PBS) (typically 3 ml). The total volume of fibers was then calculated via a conservation of volume equation: total resulting solution volume = volume of fibers + volume of PBS (3 ml). After calculating total fiber volume, solutions were re-centrifuged, supernatant was removed, and fiber pellets were resuspended to create a 10 volume % fiber solution, which were then aliquoted and stored at 4C. To support cell adhesion, 2.0 mM RGD was coupled to vinyl sulfone groups along the DexVS backbone via Michael-type addition chemistry for 30 min, followed by quenching of excess VS groups in a 300 mM cysteine solution for 30 min.

DexVS gels were formed via a thiol-ene click reaction at 3.3% (w/v) (pH 7.4, 37C, 45 min) with VPMS cross-linker (12.5, 20, and 27.5 mM) (GCRDVPMSMRGGDRCG, GenScript, George Town, KY) in the presence of varying amounts of arginylglycylaspartic acid (RGD, CGRGDS, 2.0 mM; GenScript, George Town, KY), HBP (GCGAFAKLAARLYRKA, 1.0 mM; GenScript, George Town, KY), and fiber segments (0.0 to 5.0%, v/v). For experiments comparing hydrogels of varying ligand type (1 mM HBP versus 2 mM RGD), cysteine was added to precursor solutions to maintain a final vinyl sulfone concentration of 60 mM. All hydrogel and peptide precursor solutions were made in PBS containing 50 mM Hepes. To create fibrous hydrogels, a defined stock solution (10% v/v) of suspended fibers in PBS/Hepes was mixed into hydrogel precursor solutions before gelation. By controlling the dilution of the fiber suspension, fiber density was readily tuned within the hydrogel at a constant hydrogel weight percentage. For gel contraction experiments, DexVS was polymerized within a 5-mm-diameter polydimethylsiloxane (PDMS) gasket to ensure consistent hydrogel area on day 0.

NHLFs (University of Michigan Central Biorepository), normal human dermal fibroblasts (Lonza, Basel, Switzerland), and normal human mammary fibroblasts (Sciencal, Carlsbad, CA) were cultured in Dulbeccos modified Eagles medium containing 1% penicillin/streptomycin, l-glutamine, and 10% fetal bovine serum (Atlanta Biologicals, Flowery Branch, GA). NHLFs derived from three separate donors were used for experiments. Cells were passaged upon achieving 90% confluency at a 1:4 ratio and used for studies until passage 7. For all hydrogel studies, cells were trypsinized, counted and either encapsulated into or seeded onto 25-l hydrogels at a density of 1,000,000 cells ml1 of hydrogel, and subsequently cultured at 37C and 5% CO2 in serum-containing medium. For studies comparing 3D hydrogels to tissue culture plastic, the number of cells seeded into 2D conditions was analogous to the total cell number within hydrogel matrices. Medium was refreshed the day after encapsulation and every 2 days after. In selected experiments, recombinant human TGF-1 (5 ng/ml; PeproTech, Rocky Hill, NJ) was supplemented into the medium at 5 ng ml1. For pharmacological studies, nintedanib (50 nM; Thermo Fisher Scientific, Hampton, NH), pirfenidone (100 M; Thermo Fisher Scientific, Hampton, NH), marimastat (1.0 M), and dimethyl fumarate (100 nM) were supplemented in serum-containing medium and refreshed every 2 days.

Cultures were fixed with 4% paraformaldehyde for 30 min at room temperature. To stain the actin cytoskeleton and nuclei, samples were permeabilized in PBS solution containing Triton X-100 (5%, v/v), sucrose (10%, w/v), and magnesium chloride (0.6%, w/v); blocked in 1% bovine serum albumin (BSA); and stained simultaneously with phalloidin and 4,6-diamidino-2-phenylindole (DAPI). For immunostaining, samples were permeabilized, blocked for 8 hours in 1% (w/v) BSA, and incubated with mouse monoclonal anti-YAP antibody (1:1000; Santa Cruz Biotechnology, SC-101199), mouse monoclonal anti-fibronectin antibody (FN, 1:2000; Sigma-Aldrich, #F6140), rabbit monoclonal anti-Ki67 (1:500; Sigma-Aldrich #PIMA514520), or mouse monoclonal anti-SMA (1:2000; Sigma-Aldrich, #A2547) followed by secondary antibody for 6 hours each at room temperature with 3 PBS washes in between. High-resolution images of YAP, FN, and actin morphology were acquired with a 40 objective. Unless otherwise specified, images are presented as maximum intensity projections of 100-m Z-stacks. Hydrogel samples were imaged on a Zeiss LSM 800 laser scanning confocal microscope. SHG imaging of lung tissue was conducted on a Leica SPX8 laser scanning confocal microscope with an excitation wavelength of 820 nm and a collection window of 400 to 440 nm. Single-cell morphometric analyses (cell spread area) were performed using custom Matlab scripts with sample sizes >50 cells per group, while YAP, -SMA, Ki67, and FN immunostains were quantified on an image basis with a total of 10 frames of view. MFs were denoted as nucleated, F-actin+, -SMA+ cells. For cell density (number of nuclei) calculations, DAPI-stained cell nuclei were thresholded and counted in six separate 600 m 600 m 200 m image volumes, allowing us to calculate a total number of cells per mm3 of gel. Fiber recruitment analysis was conducted via a custom Matlab script; briefly, cell outlines were created via actin masking and total fiber fluorescence was quantified under each actin mask on a per-cell basis. A similar analysis method using Matlab was used for cell-cell junction analysis as published previously, with higher area:perimeter ratios and clusters/cell indicative as more pronounced network formation (47).

For all experiments, additional hydrogel replicates were finely minced and degraded in dextranase solution (4 IU/ml; Sigma-Aldrich) for 20 min and homogenized in buffer RLT (Qiagen, Venlo, The Netherlands), and RNA was isolated according to the manufacturers protocols. Complementary DNA (cDNA) was generated from deoxyribonuclease (DNase)free RNA and amplified, and gene expression was normalized to the housekeeping gene glyceraldehyde-3-phosphate dehydrogenase (GAPDH). Experiments were run with technical triplicates across three individual biological experiments. For a complete list of primers, see table S2.

To determine the elastic modulus of lung tissue and DexVS hydrogels, indentation tests were used with a Nanosurf FlexBio AFM (Nanosurf, Liestal, Switzerland). Samples were indented via a 5-m bead at a depth of 10 m and an indentation rate of 0.333 m/s. Resulting force-displacement curves were fit to a spherical Hertz model using AtomicJ. Poissons ratios of 0.5 and 0.4 were used for hydrogels and lung tissue, respectively.

All animal studies were approved by the Animal Care and Use Committee at the University of Michigan. Bleomycin (0.025 U; Sigma-Aldrich) was instilled intratracheally in C57BL6 mice (8 weeks of age; The Jackson Laboratory, Bar Harbor, ME, USA) on day 0. Briefly, mice were anesthetized with sodium pentobarbital, the trachea was exposed and entered with a 30-gauge needle under direct visualization, and a single 30-l injection containing 0.025 U of bleomycin (Sigma-Aldrich) diluted in normal saline was injected. Lungs were collected on day 14 for mechanical and histological analysis. For histology samples, lungs were perfused with saline and inflated with 4% paraformaldehyde, sectioned, and stained with picrosirius red. For mechanical characterization via AFM, lungs were perfused with saline, infused with OCT (optimal cutting temperature) compound (Thermo Fisher Scientific), and flash-frozen in a slurry of dry ice and ethanol. Sections were mechanically tested via AFM nanoindentation immediately upon thawing.

To characterize the inflammatory secretome associated with various DexVS-VPMS environments, medium was collected from NHLF cultures 3, 5, 7, and 9 days after encapsulation. A Luminex FlexMAP 3D (Luminex Corporation, Austin, TX) systems technology was used to measure 41 cytokines/chemokines (HCTYMAG-60 K-PX41, Milliplex, EMD Millipore Corporation) in the medium samples according to the manufacturers instructions. Total secretion was reported as the sum of all 41 analytes measured for each respective condition. Cell-secreted collagen was measured using the established colorimetric Sircol assay in hydrogels cultured with serum-free medium in the presence of ascorbic acid (25 g ml1).

The NCBI GEO database was consulted [dataset GSE47460 (GP14550)] to fetch gene expression data from 91 healthy patients and 140 patients with IPF; patients with chronic obstructive pulmonary disease and nonidiopathic fibrotic lung diseases were excluded from the analysis (48). GEO2R (www.ncbi.nlm.nih.gov/geo/geo2r/) software was used for GO term enrichment, with keywords ECM, MMP, integrin, cytoskeleton, cytokine, chemokine, and MAPK used as search terms for dataset curation (48). Noncurated datasets were composed of the top 1000 differentially expressed genes between healthy and interstitial lung disease (ILD) conditions. Differential expression was calculated on the basis of subtracting normalized expression values between diseased and healthy patients. All genes were normalized before analysis with GEO2R via a pairwise cyclic losses approach. For pathway and protein-protein enrichment analyses, a curated pathway database [Reactome (49)] and Search Tool for Retrieval of Interacting Genes/Proteins [STRING (50)] methodology were consulted, respectively. For STRING analyses, up-regulated genes within the druggable genome were focused upon. A tabulated list of top genes, pathways, and nodes can be seen in data file S1.

Statistical significance was determined by one-way analysis of variance (ANOVA) or Students t test where appropriate, with significance indicated by P < 0.05. All data are presented as means SD.

Acknowledgments: We thank E. S. White (University of Michigan) for providing patient-derived lung fibroblasts used in these studies. Funding: This work was supported, in part, by the NIH (HL124322, R35HL144481). D.L.M. and C.D.D. acknowledge financial support from the NSF Graduate Research Fellowship Program (DGE1256260). Author contributions: D.L.M. and B.M.B. conceived and supervised the project. D.L.M. designed and performed the experiments. K.M.D. and K.B.A. performed and aided in analysis of the Luminex experiments. M.R.S. and C.D.D. helped with data analysis. R.P. and M.S. aided in polymer syntheses and microfiber fabrication. I.M.L. provided equipment for and assisted in polymerase chain reaction experiments. C.A.W. and B.B.M. helped perform the animal experiments for the bleomycin-induced lung fibrosis model. All authors edited and approved the manuscript. Competing interests: The authors declare that they have no competing interests. Data and materials availability: All data needed to evaluate the conclusions in the paper are present in the paper and/or the Supplementary Materials. Additional data related to this paper may be requested from the authors.

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