A coagulation defect arising from heterozygous premature termination of tissue factor. – Physician’s Weekly

Tissue factor (TF) is the primary initiator of blood coagulation in vivo and the only blood coagulation factor for which a human genetic defect has not been described. As there are no routine clinical assays that capture the contribution of endogenous TF to coagulation initiation, the extent to which reduced TF activity contributes to unexplained bleeding is unknown. Using whole genome sequencing, we identified a heterozygous frameshift variant (p.Ser117HisfsTer10) in F3, the gene encoding TF, causing premature termination of TF (TFshort) in a woman with unexplained bleeding. Routine hematological laboratory evaluation of the proposita was normal. CRISPR-edited human induced pluripotent stem cells recapitulating the variant were differentiated into vascular smooth muscle and endothelial cells that demonstrated haploinsufficiency of TF. The variant F3 transcript is eliminated by nonsense-mediated decay. Neither overexpression nor addition of exogenous recombinant TFshort inhibited factor Xa or thrombin generation, excluding a dominant negative mechanism. F3+/- mice provide an animal model of TF haploinsufficiency and exhibited prolonged bleeding times, impaired thrombus formation, and reduced survival following major injury. Heterozygous TF deficiency is present in at least 1 in 25,000 individuals and could limit coagulation initiation in undiagnosed individuals with abnormal bleeding but a normal routine laboratory evaluation.

PubMed

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A coagulation defect arising from heterozygous premature termination of tissue factor. - Physician's Weekly

Induced Pluripotent Stem Cells (iPSCs) Market Size By Product Analysis, Application, End-Users, Regional Outlook, Competitive Strategies And Forecast…

New Jersey, United States,- Latest update on Induced Pluripotent Stem Cells (iPSCs) Market Analysis report published with extensive market research, Induced Pluripotent Stem Cells (iPSCs) Market growth analysis, and forecast by 2026. this report is highly predictive as it holds the overall market analysis of topmost companies into the Induced Pluripotent Stem Cells (iPSCs) industry. With the classified Induced Pluripotent Stem Cells (iPSCs) market research based on various growing regions, this report provides leading players portfolio along with sales, growth, market share, and so on.

The research report of the Induced Pluripotent Stem Cells (iPSCs) market is predicted to accrue a significant remuneration portfolio by the end of the predicted time period. It includes parameters with respect to the Induced Pluripotent Stem Cells (iPSCs) market dynamics incorporating varied driving forces affecting the commercialization graph of this business vertical and risks prevailing in the sphere. In addition, it also speaks about the Induced Pluripotent Stem Cells (iPSCs) Market growth opportunities in the industry.

Induced Pluripotent Stem Cells (iPSCs) Market Report covers the manufacturers data, including shipment, price, revenue, gross profit, interview record, business distribution etc., these data help the consumer know about the competitors better. This report also covers all the regions and countries of the world, which shows a regional development status, including Induced Pluripotent Stem Cells (iPSCs) market size, volume and value, as well as price data.

Induced Pluripotent Stem Cells (iPSCs) Market competition by top Manufacturers:

Induced Pluripotent Stem Cells (iPSCs) Market Classification by Types:

Induced Pluripotent Stem Cells (iPSCs) Market Size by End-user Application:

Listing a few pointers from the report:

The objective of the Induced Pluripotent Stem Cells (iPSCs) Market Report:

Cataloging the competitive terrain of the Induced Pluripotent Stem Cells (iPSCs) market:

Unveiling the geographical penetration of the Induced Pluripotent Stem Cells (iPSCs) market:

The report of the Induced Pluripotent Stem Cells (iPSCs) market is an in-depth analysis of the business vertical projected to record a commendable annual growth rate over the estimated time period. It also comprises of a precise evaluation of the dynamics related to this marketplace. The purpose of the Induced Pluripotent Stem Cells (iPSCs) Market report is to provide important information related to the industry deliverables such as market size, valuation forecast, sales volume, etc.

Major Highlights from Table of contents are listed below for quick lookup into Induced Pluripotent Stem Cells (iPSCs) Market report

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Induced Pluripotent Stem Cells (iPSCs) Market Size By Product Analysis, Application, End-Users, Regional Outlook, Competitive Strategies And Forecast...

Starter Guide to induced Pluripotent Stem Cells (iPSCs …

This post was contributed by Kusumika (Kushi) Mukherjee.

The ultimate goal in the field of regenerative medicine is to replace lost or damaged cells. Here, I will discuss the two major processes by which an adult somatic cell is converted to a different cell type for regeneration and repair and situations where one process is favored over the other.

Cell conversion happens via:

The reversal of a differentiated cell type to an undifferentiated state and then redifferentiation into the cell type of choice in vitro is known as reprogramming [1]. The process can be divided into two stages:

The dedifferentiation stage involves overexpression of four reprogramming factors- OCT4, SOX2, KLF4, and C-MYC - that induce a differentiated somatic cell to revert back to a pluripotent stage (iPSC formation) [2, 3]. The iPSCs then proliferate and redifferentiate to another cell type of choice. The four reprogramming factors can be delivered and expressed in multiple somatic cells via various methods. Some of the more common delivery methodsinclude retrovirus [2], lentivirus [4], adenovirus [5], Sendai virus [6], plasmid electroporation (episomal) [7, 8] and mRNA transfection [9]. Many of the plasmids used for these methods can be found on Addgenes stem cell page. On this page, you can also find a table with a list of methods and the species they were used in. iPSCs have now been generated from many different types of somatic cells. The goal is to use cells that can be easily isolated from donors. Apart from fibroblasts, human keratinocytes from hair pluck, peripheral blood cells, and renal epithelial cells from urine are some of the easily isolated somatic cells that have been reprogrammed to iPSCs successfully [10-12].

The next stage of reprogramming consists of redifferentiation of iPSCs into the cell type of choice. This step is sometimes also referred to as directed differentiation. Specific cell media, supplements, bioactive small molecules, and growth factors are used to control the cell fate of iPSCs and differentiate them into different cell lineages [13]. Over the last decade, many cell types have been successfully differentiated from human iPSCs. Below is a list of some of these cell types[13]:

You can find a variety of plasmids for differentiation here.

Dedifferentiation to an intermediate pluripotent state is not always obligatory in cell conversion processes [35]. Rather than reprogram cells all the way back to their most primitive pluripotent stem cell state, through transdifferentiation adult somatic cells are converted directly into a different cell type, bypassing the lengthy processes of reprogramming. The process was first observed in the regenerating lens of the newt over 100 years ago [36]. While natural transdifferentiation is rare in mammals, an example is observed in the pancreas when excess -cell damage results in the transdifferentiation of glucagon-producing -cells into insulin-producing -like-cells [37, 38].

In 1987, Davis et al. reported one of the earliest examples of transdifferentiation in vitro where treatment of mouse fibroblasts with 5-azacytidine led to their conversion into myoblasts [39]. In 2000, Ferber et al. showed for the first time that mouse liver cells could be transdifferentiated in vivo to pancreatic -like-cells with the expression of pancreatic and duodenal homoeobox gene1 (PDX1) [40]. In recent works, transdifferentiation is usually carried out by expressing transcription factors specific to the lineage of the target cell in the original somatic cells [41]. The in vivo and in vitro methods are similar except that the vectors carrying the transdifferentiation factors are directly injected into the organ of interest for in vivo transdifferentiation. Multiple cell types such as fibroblasts, hepatocytes, and pancreatic exocrine cells have been successfully transdifferentiated into neurons and -cells [40-42].

Both reprogramming and transdifferentiation convert differentiated somatic cells into another cell type. However, these two approaches differ in several ways. Below is a table listing some of critical differences (adapted from Zhou and Melton, 2008, [43]):

Overall, reprogramming is very flexible. It offers unlimited potential to produce all cell types in the body. On the other hand, only few cell types have been currently transdifferentiated successfully, limiting the utility of this process. Moreover, it is much easier to genetically modify cells during the reprogramming process as they are propagated in vitro as part of the process. This opens up a wide range of possibilities in clinical situations. In cases where the objective is to fix a disease-inducing genetic mutation in a patient, trying to transdifferentiate any of the patients cells will not alleviate the problem. The best option then would be to dedifferentiate cells from the patient in vitro then correct the damaged gene in the resulting iPSCs before differentiating the cells into the correct lineage and returning them back to the patient.

In this post, I have detailed the two major processes by which cells are converted to replenish and repair cells that are lost or damaged. Both transdifferentiation and reprogramming give researchers the ability to convert a differentiated cell to a different cell type. While transdifferentiation is suited for switching cell types between similar lineages, reprogramming is more versatile and universal.

Many thanks to our guest blogger, Kusumika (Kushi) Mukherjee.

Kusumika (Kushi) Mukherjee is the Editor ofTrends in Pharmacological Sciences,a Cell Press reviews journal. She joined Cell Press to pursue a career in science communication and publishing after completing her postdoctoral training from Massachusetts General Hospital and Harvard Medical School. Connect with her on LinkedIn @https://www.linkedin.com/in/kmukherjeephd/.

References

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3. Takahashi, K. and S. Yamanaka, Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell, 2006. 126(4): p. 663-76. PubMed PMID:16904174.

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6. Ban, H., et al., Efficient generation of transgene-free human induced pluripotent stem cells (iPSCs) by temperature-sensitive Sendai virus vectors. Proc Natl Acad Sci U S A, 2011. 108(34): p. 14234-9. PubMed PMID:21821793. PubMed Central PMCID:PMC3161531.

7. Yu, J., et al., Human induced pluripotent stem cells free of vector and transgene sequences. Science, 2009. 324(5928): p. 797-801. PubMed PMID:19325077. PubMed Central PMCID:PMC2758053.

8. Okita, K., et al., Generation of mouse induced pluripotent stem cells without viral vectors. Science, 2008. 322(5903): p. 949-53. PubMed PMID:18845712.

9. Warren, L., et al., Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell, 2010. 7(5): p. 618-30. PubMed PMID:20888316. PubMed Central PMCID:PMC3656821.

10. Aasen, T., et al., Efficient and rapid generation of induced pluripotent stem cells from human keratinocytes. Nat Biotechnol, 2008. 26(11): p. 1276-84. PubMed PMID:18931654.

11. Loh, Y.H., et al., Reprogramming of T cells from human peripheral blood. Cell Stem Cell, 2010. 7(1): p. 15-9. PubMed PMID:20621044. PubMed Central PMCID:PMC2913590.

12. Zhou, T., et al., Generation of human induced pluripotent stem cells from urine samples. Nat Protoc, 2012. 7(12): p. 2080-9. PubMed PMID:23138349.

13. Williams, L.A., B.N. Davis-Dusenbery, and K.C. Eggan, SnapShot: directed differentiation of pluripotent stem cells. Cell, 2012. 149(5): p. 1174-1174 e1. PubMed PMID:22632979.

14. Sasaki, K., et al., Robust In Vitro Induction of Human Germ Cell Fate from Pluripotent Stem Cells. Cell Stem Cell, 2015. 17(2): p. 178-94. PubMed PMID:26189426.

15. Si-Tayeb, K., et al., Highly efficient generation of human hepatocyte-like cells from induced pluripotent stem cells. Hepatology, 2010. 51(1): p. 297-305. PubMed PMID:19998274. PubMed Central PMCID:PMC2946078.

16. Zhang, D., et al., Highly efficient differentiation of human ES cells and iPS cells into mature pancreatic insulin-producing cells. Cell Res, 2009. 19(4): p. 429-38. PubMed PMID:19255591.

17. Spence, J.R., et al., Directed differentiation of human pluripotent stem cells into intestinal tissue in vitro. Nature, 2011. 470(7332): p. 105-9. PubMed PMID:21151107. PubMed Central PMCID:PMC3033971.

18. Huang, S.X., et al., Efficient generation of lung and airway epithelial cells from human pluripotent stem cells. Nat Biotechnol, 2014. 32(1): p. 84-91. PubMed PMID:24291815. PubMed Central PMCID:PMC4101921.

19. Dias, J., et al., Generation of red blood cells from human induced pluripotent stem cells. Stem Cells Dev, 2011. 20(9): p. 1639-47. PubMed PMID:21434814. PubMed Central PMCID:PMC3161101.

20. Chang, C.J., et al., Production of embryonic and fetal-like red blood cells from human induced pluripotent stem cells. PLoS One, 2011. 6(10): p. e25761. PubMed PMID:22022444. PubMed Central PMCID:PMC3192723.

21. Grigoriadis, A.E., et al., Directed differentiation of hematopoietic precursors and functional osteoclasts from human ES and iPS cells. Blood, 2010. 115(14): p. 2769-76. PubMed PMID:20065292. PubMed Central PMCID:PMC2854424.

22. Jeon, O.H., et al., Human iPSC-derived osteoblasts and osteoclasts together promote bone regeneration in 3D biomaterials. Sci Rep, 2016. 6: p. 26761. PubMed PMID:20065292. PubMed Central PMCID:PMC2854424.

23. Burridge, P.W., et al., Chemically defined generation of human cardiomyocytes. Nat Methods, 2014. 11(8): p. 855-60. PubMed PMID:24930130. PubMed Central PMCID:PMC4169698.

24. Lian, X., et al., Robust cardiomyocyte differentiation from human pluripotent stem cells via temporal modulation of canonical Wnt signaling. Proc Natl Acad Sci U S A, 2012. 109(27): p. E1848-57. PubMed PMID:22645348. PubMed Central PMCID:PMC3390875.

25. Patsch, C., et al., Generation of vascular endothelial and smooth muscle cells from human pluripotent stem cells. Nat Cell Biol, 2015. 17(8): p. 994-1003. PubMed PMID:26214132. PubMed Central PMCID:PMC4566857.

26. Maffioletti, S.M., et al., Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells. Nat Protoc, 2015. 10(7): p. 941-58. PubMed PMID:26042384.

27. Nejadnik, H., et al., Improved approach for chondrogenic differentiation of human induced pluripotent stem cells. Stem Cell Rev, 2015. 11(2): p. 242-53. PubMed PMID:25578634. PubMed Central PMCID:PMC4412587.

28. Mohsen-Kanson, T., et al., Differentiation of human induced pluripotent stem cells into brown and white adipocytes: role of Pax3. Stem Cells, 2014. 32(6): p. 1459-67. PubMed PMID:24302443.

29. Kogut, I., D.R. Roop, and G. Bilousova, Differentiation of human induced pluripotent stem cells into a keratinocyte lineage. Methods Mol Biol, 2014. 1195: p. 1-12. PubMed PMID:24510784. PubMed Central PMCID:PMC4096605.

30. Lamba, D.A., et al., Generation, purification and transplantation of photoreceptors derived from human induced pluripotent stem cells. PLoS One, 2010. 5(1): p. e8763. PubMed PMID:20098701.

31. Tang, Z.H., et al., Genetic Correction of Induced Pluripotent Stem Cells From a Deaf Patient With MYO7A Mutation Results in Morphologic and Functional Recovery of the Derived Hair Cell-Like Cells. Stem Cells Transl Med, 2016. 5(5): p. 561-71. PubMed PMID:27013738. PubMed Central PMCID:PMC4835250.

32. Ma, L., Y. Liu, and S.C. Zhang, Directed differentiation of dopamine neurons from human pluripotent stem cells. Methods Mol Biol, 2011. 767: p. 411-8. PubMed PMID:21822892.

33. Shi, Y., P. Kirwan, and F.J. Livesey, Directed differentiation of human pluripotent stem cells to cerebral cortex neurons and neural networks. Nat Protoc, 2012. 7(10): p. 1836-46. PubMed PMID:22976355.

34. Wang, S., et al., Differentiation of human induced pluripotent stem cells to mature functional Purkinje neurons. Sci Rep, 2015. 5: p. 9232. PubMed PMID:25782665. PubMed Central PMCID:PMC4363833.

35. Eguizabal, C., et al., Dedifferentiation, transdifferentiation, and reprogramming: future directions in regenerative medicine. Semin Reprod Med, 2013. 31(1): p. 82-94. PubMed PMID:23329641.

36. Jopling, C., S. Boue, and J.C. Izpisua Belmonte, Dedifferentiation, transdifferentiation and reprogramming: three routes to regeneration. Nat Rev Mol Cell Biol, 2011. 12(2): p. 79-89. PubMed PMID:21252997.

37. Merrell, A.J. and B.Z. Stanger, Adult cell plasticity in vivo: de-differentiation and transdifferentiation are back in style. Nat Rev Mol Cell Biol, 2016. 17(7): p. 413-25. PubMed PMID:26979497.

38. Thorel, F., et al., Conversion of adult pancreatic alpha-cells to beta-cells after extreme beta-cell loss. Nature, 2010. 464(7292): p. 1149-54. PubMed PMID:20364121. PubMed Central PMCID:PMC2877635.

39. Davis, R.L., H. Weintraub, and A.B. Lassar, Expression of a single transfected cDNA converts fibroblasts to myoblasts. Cell, 1987. 51(6): p. 987-1000. PubMed PMID:3690668.

40. Ferber, S., et al., Pancreatic and duodenal homeobox gene 1 induces expression of insulin genes in liver and ameliorates streptozotocin-induced hyperglycemia. Nat Med, 2000. 6(5): p. 568-72. PubMed PMID:10802714.

41. Vierbuchen, T., et al., Direct conversion of fibroblasts to functional neurons by defined factors. Nature, 2010. 463(7284): p. 1035-41. PubMed PMID:20107439. PubMed Central PMCID:PMC2829121.

42. Zhou, Q., et al., In vivo reprogramming of adult pancreatic exocrine cells to beta-cells. Nature, 2008. 455(7213): p. 627-32. PubMed PMID:18754011.

43. Zhou, Q. and D.A. Melton, Extreme makeover: converting one cell into another. Cell Stem Cell, 2008. 3(4): p. 382-8. PubMed PMID:18940730.

Additional Resources on the Addgene Blog

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Starter Guide to induced Pluripotent Stem Cells (iPSCs ...

Cell Culture Protein Surface Coating Market: In-Depth Analysis Of Industry Grow – GroundAlerts.com

The study is titled Global Cell culture protein surface coating Market Research Report, in which extensive research has been undertaken by analysts and a detailed evaluation of the global market has been provided. The report includes an in-depth, extensive study of this market in tandem with vital parameters that are likely to have an effect on the market commercialization matrix.

A highly analytical qualitative as well as quantitative evaluation of the global cell culture protein surface coating market has been covered in this report. The study evaluates the myriad aspects of this industry by taking into consideration its historical and forecast data. In the research report, substantial details about Porters five force model, alongside a SWOT analysis as well as a PESTEL analysis of the market are also provided.

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The main intent of this report is to list down numerous updates and data with respect to the market and also to note the various growth opportunities that are likely to help the market expand at an appreciable rate. An in-depth synopsis of the cell culture protein surface coating market as well as a well-detailed set of market definitions and overview of the industry have been provided in the report.

The abstract section includes information on the cell culture protein surface coating market dynamics. This is further inclusive of the drivers boosting the market growth, market restraints, trends defining the industry, as well as the many growth opportunities prevailing in the industry. Also, details on the pricing analysis in tandem with the value chain analysis have been provided in the study. Historic figures and estimates with respect to the growth of this market throughout the forecast period have been entailed in the study.

The cell culture protein surface coating market report consists of information related to the projected CAGR of the global industry over the forecast period. Also, the numerous technological developments and innovations that are likely to drive the global market share over the anticipated period are mentioned in the study.

Top Companies

Split by Protein Source, the market has been divided into Animal-derived protein, Human-derived protein, Synthetic protein, Plant-derived protein

Animal-derived protein segment is anticipated to witness vigorous growth during the analysis timeframe. Animal derived protein contains high levels of heme iron, vitamin B-12, and saturated fat as well as higher levels of cholesterol augmenting segmental growth. Moreover, animal protein provides muscle health such as lean mass and strength in the quadriceps that further fosters the segmental growth.

Split by Type of Coating, the cell culture protein surface coating market has been divided into Self-coating, Pre-coating, Microwell plates, Petri dish, Flask, Slides

Self-coating segment will grow substantially during the forecast timeline owing to rising investment in research and development. Several biopharmaceutical and biotechnology companies aims on the production of protein therapeutics, production of monoclonal antibody, induced pluripotent stem cells research, cell-based assays development and cryobanking. Above mentioned factors fosters the overall segmental growth.

The regional segmentation covers

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Cell Culture Protein Surface Coating Market: In-Depth Analysis Of Industry Grow - GroundAlerts.com

Induced Pluripotent Stem Cells Market Size By Product Analysis, Application, End-Users, Regional Outlook, Competitive Strategies And Forecast Up To…

New Jersey, United States,- Latest update on Induced Pluripotent Stem Cells Market Analysis report published with extensive market research, Induced Pluripotent Stem Cells Market growth analysis, and forecast by 2026. this report is highly predictive as it holds the overall market analysis of topmost companies into the Induced Pluripotent Stem Cells industry. With the classified Induced Pluripotent Stem Cells market research based on various growing regions, this report provides leading players portfolio along with sales, growth, market share, and so on.

The research report of the Induced Pluripotent Stem Cells market is predicted to accrue a significant remuneration portfolio by the end of the predicted time period. It includes parameters with respect to the Induced Pluripotent Stem Cells market dynamics incorporating varied driving forces affecting the commercialization graph of this business vertical and risks prevailing in the sphere. In addition, it also speaks about the Induced Pluripotent Stem Cells Market growth opportunities in the industry.

Induced Pluripotent Stem Cells Market Report covers the manufacturers data, including shipment, price, revenue, gross profit, interview record, business distribution etc., these data help the consumer know about the competitors better. This report also covers all the regions and countries of the world, which shows a regional development status, including Induced Pluripotent Stem Cells market size, volume and value, as well as price data.

Induced Pluripotent Stem Cells Market competition by top Manufacturers:

Induced Pluripotent Stem Cells Market Classification by Types:

Induced Pluripotent Stem Cells Market Size by End-user Application:

Listing a few pointers from the report:

The objective of the Induced Pluripotent Stem Cells Market Report:

Cataloging the competitive terrain of the Induced Pluripotent Stem Cells market:

Unveiling the geographical penetration of the Induced Pluripotent Stem Cells market:

The report of the Induced Pluripotent Stem Cells market is an in-depth analysis of the business vertical projected to record a commendable annual growth rate over the estimated time period. It also comprises of a precise evaluation of the dynamics related to this marketplace. The purpose of the Induced Pluripotent Stem Cells Market report is to provide important information related to the industry deliverables such as market size, valuation forecast, sales volume, etc.

Major Highlights from Table of contents are listed below for quick lookup into Induced Pluripotent Stem Cells Market report

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Market Research Intellect provides syndicated and customized research reports to clients from various industries and organizations with the aim of delivering functional expertise. We provide reports for all industries including Energy, Technology, Manufacturing and Construction, Chemicals and Materials, Food and Beverage, and more. These reports deliver an in-depth study of the market with industry analysis, the market value for regions and countries, and trends that are pertinent to the industry.

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View original post here:
Induced Pluripotent Stem Cells Market Size By Product Analysis, Application, End-Users, Regional Outlook, Competitive Strategies And Forecast Up To...

Fate Therapeutics Announces FDA Clearance of IND Application for First-ever iPSC-derived CAR T-Cell Therapy | 2020-07-09 | Press Releases – Stockhouse

FT819 CAR T-cell Product Candidate Derived from Clonal Master iPSC Line with Novel CD19-specific 1XX CAR Integrated into TRAC Locus

Phase 1 Clinical Study will Evaluate FT819 for Patients with Advanced B-cell Leukemias and Lymphomas

SAN DIEGO, July 09, 2020 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, announced today that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for FT819, an off-the-shelf allogeneic chimeric antigen receptor (CAR) T-cell therapy targeting CD19+ malignancies. FT819 is the first-ever CAR T-cell therapy derived from a clonal master induced pluripotent stem cell (iPSC) line, and is engineered with several first-of-kind features designed to improve the safety and efficacy of CAR T-cell therapy. The Company plans to initiate clinical investigation of FT819 for the treatment of patients with relapsed / refractory B-cell malignancies, including chronic lymphocytic leukemia (CLL), acute lymphoblastic leukemia (ALL), and non-Hodgkin lymphoma (NHL).

The clearance of our IND application for FT819 is a ground-breaking milestone in the field of cell-based cancer immunotherapy. Our unique ability to produce CAR T cells from a clonal master engineered iPSC line creates a pathway for more patients to gain timely access to therapies with curative potential,” said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. Four years ago, we first set out under our partnership with Memorial Sloan Kettering led by Dr. Michel Sadelain to improve on the revolutionary success of patient-derived CAR T-cell therapy and bring an off-the-shelf paradigm to patients, and we are very excited to advance FT819 into clinical development.”

FT819 was designed to specifically address several limitations associated with the current generation of patient- and donor-derived CAR T-cell therapies. Under a collaboration with Memorial Sloan Kettering Cancer Center (MSK) led by Michel Sadelain, M.D., Ph.D., Director, Center for Cell Engineering, and Head, Gene Expression and Gene Transfer Laboratory at MSK, the Company incorporated several first-of-kind features into FT819 including:

The multi-center Phase 1 clinical trial of FT819 is designed to determine the maximum tolerated dose of FT819 and assess its safety and clinical activity in up to 297 adult patients across three types of B-cell malignancies (CLL, ALL, and NHL). Each indication will enroll independently and evaluate three dose-escalating treatment regimens: Regimen A as a single dose of FT819; Regimen B as a single dose of FT819 with IL-2 cytokine support; and Regimen C as three fractionated doses of FT819. For each indication and regimen, dose-expansion cohorts of up to 15 patients may be enrolled to further evaluate the clinical activity of FT819.

At the American Association for Cancer Research (AACR) Virtual 2020 Meeting, the Company presented preclinical data demonstrating FT819 is comprised of CD8a T cells with uniform 1XX CAR expression and complete elimination of endogenous TCR expression. Additionally, data from functional assessments showed FT819 has antigen-specific cytolytic activity in vitro against CD19-expressing leukemia and lymphoma cell lines that is comparable to that of healthy donor-derived CAR T cells, and persists and maintains tumor clearance in the bone marrow in an in vivo disseminated xenograft model of lymphoblastic leukemia.

Fate Therapeutics has an exclusive license for all human therapeutic use to U.S. Patent No. 10,370,452 pursuant to its license agreement with MSK1, which patent covers compositions and uses of effector T cells expressing a CAR, where such T cells are derived from a pluripotent stem cell including an iPSC. In addition to the patent rights licensed from MSK, the Company owns an extensive intellectual property portfolio that broadly covers compositions and methods for the genome editing of iPSCs using CRISPR and other nucleases, including the use of CRISPR to insert a CAR in the TRAC locus for endogenous transcriptional control.

1 Fate Therapeutics has licensed intellectual property from MSK on which Dr. Sadelain is an inventor. As a result of the licensing arrangement, MSK has financial interests related to Fate Therapeutics.

About Fate Therapeutics’ iPSC Product Platform The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 300 issued patents and 150 pending patent applications.

About Fate Therapeutics, Inc. Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for cancer and immune disorders. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Company’s immuno-oncology product candidates include natural killer (NK) cell and T-cell cancer immunotherapies, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens with chimeric antigen receptors (CARs). The Company’s immuno-regulatory product candidates include ProTmune, a pharmacologically modulated, donor cell graft that is currently being evaluated in a Phase 2 clinical trial for the prevention of graft-versus-host disease, and a myeloid-derived suppressor cell immunotherapy for promoting immune tolerance in patients with immune disorders. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking Statements This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the advancement of and plans related to the Company's product candidates and clinical studies, the Company’s progress, plans and timelines for the clinical investigation of its product candidates, the therapeutic potential of the Company’s product candidates including FT819, and the Company’s clinical development strategy for FT819. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk of difficulties or delay in the initiation of any planned clinical studies, or in the enrollment or evaluation of subjects in any ongoing or future clinical studies, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials or to support regulatory approval, difficulties in manufacturing or supplying the Company’s product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), the risk that results observed in preclinical studies of FT819 may not be replicated in ongoing or future clinical trials or studies, and the risk that FT819 may not produce therapeutic benefits or may cause other unanticipated adverse effects. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Company’s periodic filings with the Securities and Exchange Commission, including but not limited to the Company’s most recently filed periodic report, and from time to time in the Company’s press releases and other investor communications. Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Contact: Christina Tartaglia Stern Investor Relations, Inc. 212.362.1200 christina@sternir.com

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Fate Therapeutics Announces FDA Clearance of IND Application for First-ever iPSC-derived CAR T-Cell Therapy | 2020-07-09 | Press Releases - Stockhouse

Stem Cell-Derived Cells Market is Projected to Reach US$XX by the end of 2019 2029 – 3rd Watch News

Global Stem Cell-Derived Cells market Research presents a Comprehensive scenario Which can be segmented according to producers, product type, applications, and areas. This segmentation will provide deep-dive analysis of the Stem Cell-Derived Cells business for identifying the growth opportunities, development tendencies and factors limiting the development of the marketplace. This report features forecast market information based on previous and present Stem Cell-Derived Cells industry scenarios and growth facets. Each of the Essential regions coated in Stem Cell-Derived Cells report are North America, Europe, Asia-Pacific, South America, Middle East and Africa. The Stem Cell-Derived Cells market share and market prognosis of every region from 2020-2027 are presented within this report. A deep study of Stem Cell-Derived Cells marketplace dynamics will help the market aspirants in identifying the business opportunities that will lead to accumulation of earnings. This segment can efficiently determine the Stem Cell-Derived Cells hazard and key market driving forces.

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The Stem Cell-Derived Cells report is segmented to provide a clear and Precise view of this international Stem Cell-Derived Cells market statistics and market quotes. Stem Cell-Derived Cells report Information represented in the form of graphs, charts, and statistics will show the Stem Cell-Derived Cells growth rate, volume, goal customer analysis. This report presents the significant data to all Stem Cell-Derived Cells business aspirants which will facilitate useful business decisions.

key players in stem cell-derived cells market are focused on generating high-end quality cardiomyocytes as well as hepatocytes that enables end use facilities to easily obtain ready-made iPSC-derived cells. As the stem cell-derived cells market registers a robust growth due to rapid adoption in stem cellderived cells therapy products, there is a relative need for regulatory guidelines that need to be maintained to assist designing of scientifically comprehensive preclinical studies. The stem cell-derived cells obtained from human induced pluripotent stem cells (iPS) are initially dissociated into a single-cell suspension and later frozen in vials. The commercially available stem cell-derived cell kits contain a vial of stem cell-derived cells, a bottle of thawing base and culture base.

The increasing approval for new stem cell-derived cells by the FDA across the globe is projected to propel stem cell-derived cells market revenue growth over the forecast years. With low entry barriers, a rise in number of companies has been registered that specializes in offering high end quality human tissue for research purpose to obtain human induced pluripotent stem cells (iPS) derived cells. The increase in product commercialization activities for stem cell-derived cells by leading manufacturers such as Takara Bio Inc. With the increasing rise in development of stem cell based therapies, the number of stem cell-derived cells under development or due for FDA approval is anticipated to increase, thereby estimating to be the most prominent factor driving the growth of stem cell-derived cells market. However, high costs associated with the development of stem cell-derived cells using complete culture systems is restraining the revenue growth in stem cell-derived cells market.

The global Stem cell-derived cells market is segmented on basis of product type, material type, application type, end user and geographic region:

Segmentation by Product Type

Segmentation by End User

The stem cell-derived cells market is categorized based on product type and end user. Based on product type, the stem cell-derived cells are classified into two major types stem cell-derived cell kits and accessories. Among these stem cell-derived cell kits, stem cell-derived hepatocytes kits are the most preferred stem cell-derived cells product type. On the basis of product type, stem cell-derived cardiomyocytes kits segment is projected to expand its growth at a significant CAGR over the forecast years on the account of more demand from the end use segments. However, the stem cell-derived definitive endoderm cell kits segment is projected to remain the second most lucrative revenue share segment in stem cell-derived cells market. Biotechnology and pharmaceutical companies followed by research and academic institutions is expected to register substantial revenue growth rate during the forecast period.

North America and Europe cumulatively are projected to remain most lucrative regions and register significant market revenue share in global stem cell-derived cells market due to the increased patient pool in the regions with increasing adoption for stem cell based therapies. The launch of new stem cell-derived cells kits and accessories on FDA approval for the U.S. market allows North America to capture significant revenue share in stem cell-derived cells market. Asian countries due to strong funding in research and development are entirely focused on production of stem cell-derived cells thereby aiding South Asian and East Asian countries to grow at a robust CAGR over the forecast period.

Some of the major key manufacturers involved in global stem cell-derived cells market are Takara Bio Inc., Viacyte, Inc. and others.

The report covers exhaustive analysis on:

Regional analysis includes

Report Highlights:

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The Stem Cell-Derived Cells report cover following data points:

Part 1: This part enlists the global Stem Cell-Derived Cells marketplace Overview, covering the simple market debut, market analysis by kind, applications, and areas. Stem Cell-Derived Cells industry states and prognosis (2020-2027) is presented in this part. Additionally, Stem Cell-Derived Cells market dynamics saying the chances, market risk, and key driving forces are studied.

Part 2: This part covers Stem Cell-Derived Cells manufacturers profile based On their small business overview, product type, and application. Additionally, the sales volume, Stem Cell-Derived Cells product price, gross margin analysis, and Stem Cell-Derived Cells market share of every player is profiled in this report.

Part 3 and Part 4: This part presents the Stem Cell-Derived Cells competition Based on earnings, earnings, and market share of each producer. Part 4 covers the Stem Cell-Derived Cells market scenario based on regions. Region-wise Stem Cell-Derived Cells sales and growth (2015-2019) is studied in this report.

America and Europes Stem Cell-Derived Cells industry by countries. Under this Stem Cell-Derived Cells revenue, market share of those nations like USA, Canada, and Mexico is provided. Under Europe Stem Cell-Derived Cells report contains, the countries such as Germany, UK, France, Russia, Italy, Russia and their sales and growth is coated.

Part 7, Part 8 and Part 9: These 3 sections covers Stem Cell-Derived Cells The earnings and expansion in these regions are presented in this Stem Cell-Derived Cells industry report.

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Part 10 and Part 11: This component depicts the Stem Cell-Derived Cells marketplace Share, earnings, sales by product type and application. The Stem Cell-Derived Cells sales growth seen during 2012-2020 is covered in this report.

Related to Stem Cell-Derived Cells market (2020-2027) for every region. The sales channels including indirect and direct Stem Cell-Derived Cells advertising, traders, distributors, and future trends are presented in this report.

Part 14 and Part 15: These components present Stem Cell-Derived Cells market key Research findings and judgment, research methodology, and data sources are covered.

Therefore, Global Stem Cell-Derived Cells report is a complete blend covering all The very important market aspects.

Continued here:
Stem Cell-Derived Cells Market is Projected to Reach US$XX by the end of 2019 2029 - 3rd Watch News

Could induced pluripotent stem cells be the breakthrough …

Embryonic stem cells. The ethical issues associated with stem cell research could be resolved through the use of induced pluripotent stem cells, which are derived from fully committed and differentiated cells of the adult body

The almost miraculous benefits that stem cells may one day deliver have long been speculated on. Capable of becoming different types of cells, they offer huge promise in terms of transplant and regenerative medicine. It is, however, also a medical field that urges caution one that must constantly battle exaggeration. If stem cells do in fact hold the potential to reverse the ageing process, for example, then such breakthroughs remain many years away.

Recently, though, the field has had cause for excitement. In 2006, Japanese researcher Shinya Yamanaka discovered that mature cells could be reprogrammed to become pluripotent, meaning they can give rise to any cell type of the body. In 2012, the discovery of these induced pluripotent stem cells (iPSCs) saw Yamanaka and British biologist John Gurdon awarded the Nobel Prize in Physiology or Medicine. Since then, there has been much talk regarding the potential iPSCs possess, not only for the world of medicine, but for society more generally, too.

A big stepHistorically, one of the major hurdles preventing further research into stem cells has been an ethical one. Until the discovery of iPSCs, embryonic stem cells (ESCs) represented the predominant area of research, with cells being taken from preimplantation human embryos. This process, however, involves the destruction of the embryo and, therefore, prevents the development of human life. Due to differences in opinion over when life is said to begin during embryonic development, stem cell researchers face an ethical quandary.

The promise of significant health benefits and new revenue streams has led some clinics to offer unproven stem cell treatments to individuals

With iPSCs, though, no such dilemmas exist. IPSCs are almost identical to ESCs but are derived from fully committed and differentiated cells of the adult body, such as a skin cell. Like ESCs, iPSCs are pluripotent and, as they are stem cells, can self-renew and differentiate, remaining indefinitely propagated and retaining the ability to give rise to any human cell type over time.

One important distinction to make is that both ESCs and iPSCs do not exist in nature, Vittorio Sebastiano, Assistant Professor (Research) of Obstetrics and Gynaecology (Reproductive and Stem Cell Biology) at Stanford Universitys Institute for Stem Cell Biology and Regenerative Medicine, told The New Economy. They are both beautiful laboratory artefacts. This means that at any stage of development, you cannot find ESCs or iPSCs in the developing embryo, foetus or even in the postnatal or adult body. Both ESCs and iPSCs can only be established and propagated in the test tube.

The reason neither ESCs nor iPSCs can be found in the body is that they harbour the potential to be very dangerous. As Sebastiano explained, these cells could spontaneously differentiate into tumorigenic masses because of their intrinsic ability to give rise to any cell type of the body. Over many years of research, scientists have learned how to isolate parts of the embryo (in the case of ESCs) and apply certain culture conditions that can lock cells in their proliferative and stem conditions. The same is true for iPSCs.

To create iPSCs, scientists take adult cells and exogenously provide a cocktail of embryonic factors, known as Yamanaka factors, for a period of two to three weeks. If the expression of such factors is sustained for long enough, they can reset the programme of the adult cells and establish an embryonic-like programme.

Turning back the clockThere is already a significant body of research dedicated to how stem cells can be used to treat disease. For example, mesenchymal stem cells (usually taken from adult bone marrow) have been deployed to treat bone fractures or as treatments for autoimmune diseases. It is hoped that iPSCs could hold the key for many more treatments.

Global stem cell market:25.5%Expected compound annual growth rate (2018-24)$467bnExpected market value (2024)

IPSCs are currently utilised to model diseases in vitro for drug screening and to develop therapies that one day will be implemented in people, Sebastiano explained. Given their ability to differentiate into any cell type, iPSCs can be used to differentiate into, for example, neurons or cardiac cells, and study specific diseases. In addition, once differentiated they can be used to test drugs on the relevant cell type. Some groups and companies are developing platforms for cell therapy, and I am personally involved in two projects that will soon reach the clinical stage.

Perhaps the most exciting prospects draw on iPSCs regenerative properties. Over time, cells age for a variety of reasons namely, increased oxidative stress, inflammation and exposure to pollutants or sunlight, among others. All these inputs lead to an accumulation of epigenetic mistakes those that relate to gene expression rather than an alteration of the genetic code itself in the cells, which, over time, results in the aberrant expression of genes, dysfunctionality at different levels, reduced mitochondrial activity, senescence and more besides. Although the epigenetic changes that occur with time may not be the primary cause of ageing, the epigenetic landscape ultimately affects and controls cell functionality.

What we have shown is that, if instead of being expressed for two weeks we express the reprogramming factors for a very short time, then we see that the cells rejuvenate without changing their identity, Sebastiano said. In other words, if you take a skin cell and express the reprogramming genes for two to four days, what you get is a younger skin cell.

By reprogramming a cell into an iPSC, you end up with an embryonic-like cell the reprogramming erases any epigenetic errors. If expressed long enough, it erases the epigenetic information of cell identity, leaving embryonic-like cells that are also young.

Slow and steadyAs with any scientific advancement, financial matters are key. According to Market Research Engine, the global stem cell market is expected to grow at a compound annual growth rate of 25.5 percent between 2018 and 2024, eventually reaching a market value of $467bn. The emergence of iPSCs has played a significant role in shaping these predictions, with major bioscience players, such as Australias Mesoblast and the US Celgene, working on treatments involving this particular type of stem cell.

The business potential around stem cell research is huge, Sebastiano told The New Economy. [Particularly] when it comes to developing cell banks for which we have detailed genetic information and, for example, studying how different drugs are toxic or not on certain genetic backgrounds, or when specific susceptibility mutations are present.

Unfortunately, even as the business cases for iPSC treatments increase, a certain degree of caution must be maintained. The promise of significant health benefits and new revenue streams has led some clinics to offer unproven stem cell treatments to individuals. There have been numerous reports of complications emerging, including the formation of a tumour following experimental stem cell treatment in one particular patient, as recorded in the Canadian Medical Association Journal last year. Such failures risk setting the field back years.

The challenge for researchers now will be one of balance. The potential of iPSCs is huge both in terms of medical progress and business development but can easily be undermined by misuse. Medical advancements, particularly ones as profound as those associated with iPSCs, simply cannot be rushed.

Read more here: Could induced pluripotent stem cells be the breakthrough genetics has been waiting for? - The New Economy

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Could induced pluripotent stem cells be the breakthrough ...

Global Induced Pluripotent Stem Cells Market 2020-2026 Demand and Insights Analysis Report – Cole of Duty

The market study on the global Induced Pluripotent Stem Cells market will encompass the entire ecosystem of the industry, covering major regions namely North America, Europe, Asia Pacific, South America, Middle East & Africa, and the major countries falling under those regions. The study will feature estimates in terms of sales revenue and consumption from 2020 to 2026, at the global level and across the major regions mentioned above. The study has been created using a unique research methodology specifically designed for this market.

Quantitative information includes Induced Pluripotent Stem Cells market estimates and forecast for a upcoming years, at the global level, split across the key segments covered under the scope of the study, and the major regions and countries. Sales revenue and consumption estimates, year-on-year growth analysis, price estimation and trend analysis, etc. will be a part of quantitative information for the mentioned segments and regions/countries.

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Qualitative information will discuss the key factors driving the restraining the growth of the market, and the possible growth opportunities of the market, regulatory scenario, value chain and supply chain analysis, export and import analysis, attractive investment proposition, and Porters 5 Forces analysis among others will be a part of qualitative information. Further, justification for the estimates for each segments, and regions will also be provided in qualitative form.

Major Points covered in this report are as below

Major players profiled in the report include The Fujifilm Holding Corporation, Astellas Pharma, Fate Therapeutics, Bristol-Myers Squibb Company, ViaCyte, Celgene Corporation, Aastrom Biosciences, Acelity Holdings, StemCells, Japan Tissue Engineering, Organogenesis,.

The study will also feature the key companies operating in the industry, their product/business portfolio, market share, financial status, regional share, segment revenue, SWOT analysis, key strategies including mergers and acquisitions, product developments, joint ventures and partnerships an expansions among others, and their latest news as well. The study will also provide a list of emerging players in the Induced Pluripotent Stem Cells market.

Based on regions, the market is classified into North America, Europe, Asia Pacific, Middle East and Africa and Latin America. The study will provide detailed qualitative and quantitative information on the above mentioned segments for every region and country covered under the scope of the study.

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Global Induced Pluripotent Stem Cells Market 2020-2026 Demand and Insights Analysis Report - Cole of Duty

Induced Pluripotent Stem Cells Market Size |Incredible Possibilities and Growth Analysis and Forecast To 2025 – AlgosOnline

Induced Pluripotent Stem Cells Market Size |Incredible Possibilities and Growth Analysis and Forecast To 2025 Published: 22 hours ago Author: Partha Ray Category: #industry

Global Induced Pluripotent Stem Cells Market Report estimates the drivers, restraints, and opportunities pertaining to the Induced Pluripotent Stem Cells industry over the timeframe of 2019-2024. Delivering the key insights pertaining to this industry, the report provides an in-depth analysis of the latest trends, present and future business scenario, market size and share of Induced Pluripotent Stem Cells industry over the coming five years.

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The Induced Pluripotent Stem Cells research report provides a detailed assessment of this business sphere. This report also estimates the market share and growth rate attained over the forecast period. The report unravels all the key aspects of Induced Pluripotent Stem Cells market including revenue forecasts, industry size, and sales amassed with respected to each industry segment. The key growth drivers and the restraints of this industry vertical have also been elucidated in the report.

Understanding the Induced Pluripotent Stem Cells market with respect to the regional landscape:

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Additional insights of the Induced Pluripotent Stem Cells market report are listed below:

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Induced Pluripotent Stem Cells Regional Market Analysis

Induced Pluripotent Stem Cells Segment Market Analysis (by Type)

Induced Pluripotent Stem Cells Segment Market Analysis (by Application)

Induced Pluripotent Stem Cells Major Manufacturers Analysis

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