X4 Pharmaceuticals Appoints Renato Skerlj, Ph.D. as Senior Vice President of Research and Development – Business Wire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, today announced the appointment of Renato Skerlj, Ph.D., as Senior Vice President, Research and Development. Dr. Skerlj has twenty-five years of experience leading the discovery and development of small molecule drugs to treat rare diseases, cancer, infection and neurodegenerative diseases. In addition, he was one of the original founders of X4 Pharmaceuticals.

Renatos deep scientific expertise in the research and development of innovative, genetically-targeted treatments, combined with his foundational knowledge of X4 and our novel CXCR4 platform, will be invaluable as we advance our pre-clinical product candidates and further expand our rare disease pipeline, said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. We are thrilled to have Renato join our senior leadership team given his instrumental role in the founding of X4.

I am very pleased to become a part of X4s dedicated team of experienced and thoughtful leaders, especially during this exciting period of X4s R&D expansion and corporate growth. I look forward to leading our Vienna-based research team and applying my decades of strategic scientific experience to advance and deliver novel therapeutic options to patients with rare diseases, commented Dr. Skerlj.

Most recently, Dr. Skerlj held drug discovery and development leadership roles at Cambridge-based Lysosomal Therapeutics, Inc. Prior to that, he was interim Head of Small Molecule Discovery at Genzyme, and was part of the executive team at AnorMED, a publicly-traded company that was acquired by Genzyme in 2006. Dr. Skerlj is an inventor of both plerixafor, a stem cell mobilizer approved by the U.S. Food and Drug Administration (FDA) in 2008, and ertapenem, an anti-bacterial approved by the FDA in 2001, and has been responsible for delivering multiple drug candidates into early clinical research. He has authored 65 publications and holds 50 patents. Dr. Skerlj received his Ph.D. in Synthetic Organic Chemistry from the University of British Columbia and completed postdoctoral fellowships at the University of Oxford and Ohio State University.

About X4 Pharmaceuticals

X4 Pharmaceuticals is developing novel therapeutics designed to improve immune cell trafficking to treat rare diseases, including primary immunodeficiencies and certain cancers. The companys oral small molecule drug candidates antagonize the CXCR4 pathway, which plays a central role in immune surveillance. X4s most advanced product candidate, mavorixafor (X4P-001), is in a global Phase 3 pivotal trial in patients with WHIM syndrome, a rare, inherited, primary immunodeficiency disease, and is currently also under investigation in combination with axitinib in the Phase 2a portion of an open-label Phase 1/2 clinical trial in clear cell renal cell carcinoma (ccRCC). X4 is also planning to commence clinical trials of mavorixafor in Severe Congenital Neutropenia (SCN) and Waldenstrms macroglobulinemia (WM) in 2019. X4 was founded and is led by a team with extensive biopharmaceutical product development and commercialization expertise and is committed to advancing the development of innovative medicines on behalf of patients with limited treatment options. X4 is a global company that is headquartered in Cambridge, Massachusetts with research offices based in Vienna, Austria. For more information, please visit http://www.x4pharma.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. The words may, will, could, would, should, expect, plan, anticipate, intend, believe, estimate, predict, project, potential, continue, target and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements include, but are not limited to, statements regarding X4s business strategy, including its clinical development plans. These statements are subject to various risks and uncertainties, actual results could differ materially from those projected, and X4 cautions investors not to place undue reliance on the forward-looking statements in this press release. These risks and uncertainties include, without limitation, the risk that any one or more of X4s product candidates will not be successfully developed, approved or commercialized, the risk that X4s ongoing or planned clinical trials and studies may be delayed, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving X4s product candidates and the risk that costs required to develop X4s product candidates or to expand its operations will be higher than anticipated. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, the risks and uncertainties described in the section entitled Risk Factors in X4s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as updated by X4s Current Report on Form 8-K filed with the SEC on April 11, 2019, and in other filings X4 makes with the SEC from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances.

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X4 Pharmaceuticals Appoints Renato Skerlj, Ph.D. as Senior Vice President of Research and Development - Business Wire

Is Stem Cell Therapy for Arthritis Safe and Effective?

People considering stem cell treatment for arthritis want to know Is it safe? and Is it effective?

Most stem cell therapy using adult stem cells is considered safe because the stem cells are collected from the patient, minimizing the risk of an unwanted reaction. The most common side effects are temporary swelling and pain.1

While most stem cell therapy for arthritis is considered safe, it does carry the same risks as any other medical procedure, such as a small risk of infection. Risk may be increased if:

See What Are Stem Cells?

Some research suggests stem cell therapy engaging in these kinds of practices may elevate the risk of tumors.2

As with most regenerative medicine treatments, research is ongoing, and FDA regulations are relatively new and subject to change.

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Whether or not stem cells therapy is effective in treating osteoarthritis is a controversial subject among medical professionals, and research in the area is ongoing.

See Osteoarthritis Treatment

How researchers think stem cell therapy worksResearchers theorize3 that when applied to an arthritic joint, stem cells might:

See Osteoarthritis Symptoms and Signs

It may be none, one, two, or all three processes at are work.

Proponents vs criticsLike many relatively new treatments, stem cell therapy has proponents and critics.

Critics emphasize that there have been no large-scale, prospective, double-blind research studiesthe kind of clinical studies that medical professionals consider the gold standardto support stem cell therapy for arthritis.

Factors that affect stem cell therapy researchAnother challenge associated with current stem cell research is that there is no standard stem cell therapy for arthritis treatment. So the stem cell therapy in one study is not necessarily the same as the stem cell therapy in another study.

Differences can include:

These differences are further complicated by more unknowns. For example, how many stem cells are needed for a particular treatment? And how do we determine if a patients own stem cells are competent enough to aid in healing?

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Many physicians combine the use of stem cells with platelet rich plasma, or PRP.

See Platelet-Rich Plasma (PRP) Therapy for Arthritis

PRP is derived from a sample of the patients blood. In the body, platelets secrete substances called growth factors and other proteins that regulate cell division, stimulate tissue regeneration, and promote healing. Like stem cell therapy, PRP therapy is sometimes used alone with the hopes of healing an arthritic joint.

See PRP Injection Preparation and Composition

Physicians who use PRP and stem cells together think that the PRP can help maximize the healing effects of stem cells.6,7 Research in this area is ongoing.

See Platelet-Rich Plasma Injection Procedure

Stem cell therapy can vary depending on the doctor performing it. People considering stem cell therapy for an arthritic knee or other joint are advised to ask their doctors questions, including:

Both doctors and patients can benefit from having a frank conversation and setting reasonable expectations.

See Arthritis Treatment Specialists

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Is Stem Cell Therapy for Arthritis Safe and Effective?

Clinical trial of stem cell therapy for traumatic spinal …

April 27, 2018

Mayo Clinic is enrolling patients in a phase 1 clinical trial of adipose stem cell treatment for spinal cord injury caused by trauma. The researchers already have approval from the Food and Drug Administration for subsequent phase 2A and 2B randomized control crossover trials.

Participants in the phase 1 clinical trial must have experienced a trauma-related spinal cord injury from two weeks to one year prior to enrollment. They will receive intrathecal injections of adipose-derived mesenchymal stem cells. No surgery or implantable medical device is required.

"That is the most encouraging part of this study," says Mohamad Bydon, M.D., a consultant in Neurosurgery specializing in spinal surgery at Mayo Clinic in Rochester, Minnesota, and the study's director. "Intrathecal injection is a well-tolerated and common procedure. Stem cells can be delivered with an implantable device, but that would require surgery for implantation and additional surgeries to maintain the device. If intrathecal treatment is successful, it could impact patients' lives without having them undergo additional surgery or maintain permanently implantable devices for the rest of their lives."

To qualify for the trial, patients must have a spinal cord injury of grade A or B on the American Spinal Injury Association (ASIA) Impairment Scale. After evaluation at Mayo Clinic, eligible patients who enroll will have adipose tissue extracted from their abdomens or thighs. The tissue will be processed in the Human Cellular Therapies Laboratories, which are co-directed by Allan B. Dietz, Ph.D., to isolate and expand stem cells.

Four to six weeks after the tissue extraction, patients will return to Mayo Clinic for intrathecal injection of the stem cells. The trial participants will then be evaluated periodically for 96 weeks.

Mayo Clinic has already demonstrated the safety of intrathecal autologous adipose-derived stem cells for neurodegenerative disease. In a previous phase 1 clinical trial, with results published in the Nov. 22, 2016, issue of Neurology, Mayo Clinic researchers found that therapy was safe for people with amyotrophic lateral sclerosis (ALS). The therapy, developed in the Regenerative Neurobiology Laboratory under the direction of Anthony J. Windebank, M.D., is moving into phase 2 clinical trials.

Dr. Windebank is also involved in the new clinical trial for people with traumatic spinal cord injuries. "We have demonstrated that stem cell therapy is safe in people with ALS. That allows us to study this novel therapy in a different population of patients," he says. "Spinal cord injury is devastating, and it generally affects people in their 20s or 30s. We hope eventually that this novel therapy will reduce inflammation and also promote some regeneration of nerve fibers in the spinal cord to improve function."

Mayo Clinic's extensive experience with stem cell research provides important guidance for the new trial. "We know from prior studies that stem cell treatment can be effective in aiding with regeneration after spinal cord injury, but many questions remain unanswered," Dr. Bydon says. "Timing of treatment, frequency of treatment, mode of delivery, and number and type of stem cells are all open questions. Our hope is that this study can help answer some of these questions."

In addition to experience, Mayo Clinic brings to this clinical trial the strength of its multidisciplinary focus. The principal investigator, Wenchun Qu, M.D., M.S., Ph.D., is a consultant in Physical Medicine and Rehabilitation at Mayo Clinic's Minnesota campus, as is another of the trial's investigators, Ronald K. Reeves, M.D. Dr. Dietz, the study's sponsor, is a transfusion medicine specialist. Also involved is Nicolas N. Madigan, M.B., B.Ch., BAO, Ph.D., a consultant in Neurology at Mayo Clinic's Minnesota campus.

The study team is in discussions with U.S. military medical centers to enroll patients, and discussing additional collaboration with international sites, potentially in Israel or Europe, for future phases of the study.

"At Mayo Clinic, we have a high-volume, patient-centered multidisciplinary practice," Dr. Bydon says. "That allows us to do the most rigorous scientific trial that is in the best interests of our patients."

Mayo Clinic. Adipose Stem Cells for Traumatic Spinal Cord Injury (CELLTOP). ClinicalTrials.gov.

Staff NP, et al. Safety of intrathecal autologous adipose-derived mesenchymal stromal cells in patients with ALS. Neurology. 2016;87:2230.

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Clinical trial of stem cell therapy for traumatic spinal ...

Stem Cell Rejuvenation Center > About Us

About Us

"We use the power of naturopathic medicine in our approach to stem cell treatment. In our logo, inside a cell is the bodhi leaf. It symbolizes healing, inner peace, health, progress and release. This enlightened care is what we bring you." -- Dr. Timothy Peace

Our Location

640 W. Maryland Ave., Suite 3Phoenix, Arizona 85013 (602) 439-0000 (602) 439-0021 info@the-stem-cell-center.com

Located in beautiful Phoenix, Arizona, we are one of the original stem cell therapy centers. With over 30 years combined experience in the stem cell field, our staff has revolutionized stem cell therapy through integrative thinking which has allowed our patients to experience long lasting and effective results. All our procedures are done on site at our clinic here in Phoenix. It is our top priority to provide you a safe, clean, sterile and friendly environment.

Our Treatment Center is located just 20 minutes from the Phoenix Sky Harbor International Airport and many hotels provide shuttle service to and from our clinic making it ideal for out-of-town visitors.

Founded in the U.S.A., we perform all therapies within the United States. Neither our patients nor the stem cells that we use are transported outside the United States. We use less than minimally manipulated technology to provide Stem Cell and PRP therapies originally initiated during the 1990's.

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Stem Cell Rejuvenation Center > About Us

How to Get the Most from Stem Cell Therapy – Medical Center

Regenerative Medicineis a fast-growing branch of medicine which focuses on regenerating damaged tissues using human cells. These cells can come from the patients own bone marrow, adipose (fat) tissue or blood, in addition to Whartons Jelly, amniotic, or umbilical cord tissue donated by healthy mothers during C-section births. These cells are injected into the body, bringing with them a host of growth factors, cytokines, and mesenchymal stem cells. The healing properties create an inflammatory response by the body while generating scaffolding and recruiting the cell types necessary to heal the damaged tissues. Regenerative Medicine is proven a viable option for healing damaged tendons, ligaments, muscles and even bone.

The two most commonly-known modalities used in Regenerative Medicine arePlatelet Rich Plasma(PRP) andStem Cell Therapy. PRP uses the growth factors within the patients own blood to provide notable healing for soft-tissue injuries. During this simple, yet effective procedure, the patients blood is drawn, treated to isolate the beneficial nutrients, and injected into the injured area(s). Austin Preferred Integrative Medicine uses this modality alongside Physical Medicine and Rehab services to help patients get the most out of their treatment plans. The focus of this article, however, will be Stem Cell Therapy.

Stem Cell Therapy, while similar in concept to PRP, is a much more advanced treatment option. This technique has generated much discussion in recent years, due to its remarkable results and wide applicability. Stem Cell Therapy can use autologous tissues (derived from the patients body) or allogeneic tissues (derived from an outside source). Both methods can produce excellent results and are even seen as a viable alternative to surgery in many cases. Austin Preferred Integrative Medicine uses aWhartons Jellyproduct which is stored in a liquid nitrogen tank, allowing us to preservelive stem cellsprior to the injection. Many other clinics offering Stem Cell Therapy in Austin cannot offer this to their patients.

Stem Cell Therapy is also being tested as a possible solution to other conditions including strokes, blood disease and diabetes, to name a few.

In addition to the superior products Austin Preferred Integrative Medicine uses for Stem Cell Therapy, we also provide our patients with an abundance of information and guidance to increase the likelihood of a great outcome. Following are some helpful pre and post-injection instructions to enhance the effectiveness of Regenerative Medicine procedures:

Refrain from taking NSAIDs (Advil, Motrin, Aleve, Celebrex, Naproxen, Mobic, etc.) for one week prior to the injection. These can interfere with the initial inflammatory response created by the stem cells. Instead, take acetaminophen if necessary.

Stay hydrated prior to your procedure. We recommend that patients drink at least 72 ounces of water per day for three days prior to the injection.

Reduce the amount of sugar, calories and triglycerides in your diet for at least one week prior to the injection.

Allow our healthcare providers to review any diagnostic imaging (MRIs, X-Rays, EMGs) to ensure your candidacy for the procedure.

While soreness should occur, it is best to continue carrying out light activities around your home, or even incorporating some low-impact walking into your daily routine. These are both better options than remaining completely sedentary, but use pain as your guideline for all activities.

Limit your use of the NSAIDs for the first six weeks after the injection. These drugs interfere with the inflammation process. Limiting them allows the body to set up the scaffolding necessary for tissue regeneration.

Consume a diet rich in lean meat, eggs, whole grains, fresh produce and healthy fats. This allows the body to optimize its internal processes, including healing.

ConsiderPhysical Therapy,Laser Therapyandsupportive bracesas options to help facilitate the recovery process.

This is just a fraction of the information Austin Preferred Integrative medicine provides to all Stem Cell patients. Unlike many other practices offering Stem Cell Therapy in Austin, we strive to work hand-in-hand with our patients. This philosophy helps our healthcare providers achieve the best results possible.

Austin Preferred also offers Free Consultations for in Stem Cell Therapy in Austin. Call (512) 442-2727 and email info@austinpreferred.com with questions or to schedule your free consultation. You can also refer to ourServicesandConditionspages for more information on treatment at our practice.

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How to Get the Most from Stem Cell Therapy - Medical Center

Platelet-Rich Plasma Injection Procedure – Sports-health

PRP injections are performed as an outpatient procedure. Because the patients blood must be drawn and prepared for injection, a typical procedure may take anywhere from 45 to 90 minutes.

An experienced physician should perform the injections. The use of imaging technology, such as ultrasound, may be used to ensure a safe and precise placement of the injection into the damaged tendon.

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The American Academy of Orthopaedic Surgeons recommends patients avoid or discontinue certain medications prior to injection:

In addition, patients are advised to drink plenty of fluids the day before the procedure. Some patients may require anti-anxiety medication immediately before the procedure.

The American Academy of Orthopaedic Surgeons does not advocate for or against platelet-rich plasma treatment.1

This is an in-office procedure that involves a blood draw, preparation of the PRP, and the injection:

The platelet-rich plasma typically stimulates a series of biological responses, including inflammation, so the injection site may be swollen and painful for about 3 to 5 days.

Platelet rich plasma injections may cause temporary inflammation, pain, and swelling. Patients are often advised to take it easy for a few days and avoid putting strain on the affected joint.

A doctor may recommend that a patient:

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If the patient does not have a physically demanding job, he or she can usually go back to work the next day. Patients can usually resume normal activities a few days after the injections, when swelling and pain decrease. Patients should not begin taking anti-inflammatory medications until approved by the doctor.

The patient will likely be prescribed post-injection physical therapy. A licensed physical therapist can teach the patient exercises that build and maintain joint strength and flexibility.

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Platelet-Rich Plasma Injection Procedure - Sports-health

What are Induced Pluripotent Stem (IPS) Cells? – Stem Cell …

However, there are a few key differences between these cells. For starters, iPS cells are not dependent on the use of cells from an embryo. Some research also indicates that some of the genes in induced pluripotent cells can behave differently compared to those in embryonic stem cells. This can be caused by an incomplete reprogramming of the cells or genetic changes acquired by the iPS cells as they multiply and grow.

Researchers still have a lot to study in terms of understanding how reprogramming works inside a cell, which is why many scientists believe iPS cells cant replace embryonic stem cells in basic research.

Though iPS cells are a large advancement for medical research, there is still a lot of research to be done in terms of understanding exactly how the reprogramming occurs in a cell. Researchers must also study iPS cells more to understand how iPS cells can be produced consistently and controlled enough to meet quality and safety requirements for the lab/clinic, as well how cells made from iPS cells will behave in the body. iPS cells behave differently from embryonic stem cells, and researchers are still looking at the effects these may have on medicine and research. There is still more to be done in terms of developing affordable and effective iPS cell treatments, as this still remains a constant challenge.

By studying iPS cells closely, scientists were able to develop a way to create these cells without making permanent genetic changes that were linked to the possible formation of tumors. Research and close studies are allowing the creation of iPS obtained specialized cells that will be safe for use for patients, but again, there is still a lot more research to be done.

IPS Cells and The Future

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What are Induced Pluripotent Stem (IPS) Cells? - Stem Cell ...

Induced Pluripotent Stem Cell – ScienceDirect

A.. Owaidah, W.. Kafienah, in Reference Module in Biomedical Sciences, 2016

Induced pluripotent stem cells are believed to be an alternative source for ESC in cell based therapies. These cells were found to share similar characteristics to ESCs in terms of unlimited self-renewal capacity and pluripotency. Their capacity to generate cartilaginous tissue was first evident in teratomas in vivo (Takahashi et al., 2007; Takahashi and Yamanaka, 2006; Boulting et al., 2011; Ellis et al., 2009; Yu et al., 2007). This suggested that iPSC exhibit the capacity to differentiate down the chondrogenic lineage. The methods reported on the derivation of chondrocytes from iPSCs were similar to those reported with ESCs, including techniques based on EB formation, co-culture methods with normal chondrocytes and direct differentiation (Guzzo et al., 2013; Wei et al., 2012; Yamashita et al., 2013; Kim et al., 2011).

Despite the fact that iPSC represent an alternative source to ESC in cell-based therapies, the genomic changes that are associated with most of the reprogramming methods hinder the progression towards clinical applications. In 2014, Borestrom and colleagues (Borestrm et al., 2014) showed that reprograming articular chondrocytes using RNA-based methods eliminates the risk of genomic integration and aberrations. In the same study, reprogrammed cells were successfully differentiated into chondrocytes using two different methods. One protocol involved the directed differentiation on monolayer method presented by Oldershaw et al in 2010. The second method involved the generation of cartilage pellets from iPSC with the supplementation of exogenous TGF-1. Chondrogenic differentiation and cartilage nodule formation were again assessed only through chondrogenic gene expression and alcian blue staining without accurate, quantitative matrix analysis.

In conclusion, although there are various methods that can be used to achieve chondrogenic differentiation of both ESCs and iPSCs, current methods only allow for the generation of small scale chondrogenic cultures without methods for competent scale up that permit large-scale tissue engineering for the repair of bona fide cartilage defects.

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Induced Pluripotent Stem Cell - ScienceDirect

Direct generation of human naive induced pluripotent stem …

Evans, M. J. & Kaufman, M. H. Establishment in culture of pluripotential cells from mouse embryos. Nature 292, 154156 (1981).

Thomson, J. A. et al. Embryonic stem cell lines derived from human blastocysts. Science 282, 11451147 (1998).

Takahashi, K. et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131, 861872 (2007).

Hackett, J. A. & Surani, M. A. Regulatory principles of pluripotency: from the ground state up. Cell Stem Cell 15, 416430 (2014).

Davidson, K. C., Mason, E. A. & Pera, M. F. The pluripotent state in mouse and human. Development 142, 30903099 (2015).

Osafune, K. et al. Marked differences in differentiation propensity among human embryonic stem cell lines. Nat. Biotechnol. 26, 313315 (2008).

Weinberger, L., Ayyash, M., Novershtern, N. & Hanna, J. H. Dynamic stem cell states: naive to primed pluripotency in rodents and humans. Nat. Rev. Mol. Cell Biol. 17, 155169 (2016).

Luni, C. et al. High-efficiency cellular reprogramming with microfluidics. Nat. Methods 13, 446452 (2016).

Zhang, J. et al. LIN28 regulates stem cell metabolism and conversion to primed pluripotency. Cell Stem Cell 19, 6680 (2016).

Takashima, Y. et al. Resetting transcription factor control circuitry toward ground-state pluripotency in. Hum. Cell 158, 12541269 (2014).

Theunissen, T. W. et al. Systematic identification of culture conditions for induction and maintenance of naive human pluripotency. Cell Stem Cell 15, 471487 (2014).

Warren, L. et al. Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell 7, 618630 (2010).

Gafni, O. et al. Derivation of novel human ground state naive pluripotent stem cells. Nature 504, 282286 (2013).

Pastor, W. A. et al. Naive human pluripotent cells feature a methylation landscape devoid of blastocyst or germline memory. Cell Stem Cell 18, 323329 (2016).

Yoshida, Y., Takahashi, K., Okita, K., Ichisaka, T. & Yamanaka, S. Hypoxia enhances the generation of induced pluripotent stem cells. Cell Stem Cell 5, 237241 (2009).

Watanabe, K. et al. A ROCK inhibitor permits survival of dissociated human embryonic stem cells. Nat Biotechnol. 6, 681686 (2007).

Theunissen, T. W. et al. Molecular criteria for defining the naive human pluripotent state. Cell Stem Cell 19, 502515 (2016).

Guo, G. et al. Naive pluripotent stem cells derived directly from isolated cells of the human inner cell mass. Stem Cell Rep. 6, 437446 (2016).

Liu, X. et al. Comprehensive characterization of distinct states of human naive pluripotency generated by reprogramming. Nat. Methods 14, 10551062 (2017).

Kilens, S. et al. Parallel derivation of isogenic human primed and naive induced pluripotent stem cells. Nat. Commun. 9, 360 (2018).

Cacchiarelli, D. et al. Integrative analyses of human reprogramming reveal dynamic nature of induced pluripotency. Cell 162, 412424 (2015).

Smith, Z. D. et al. DNA methylation dynamics of the human preimplantation embryo. Nature 511, 611 (2014).

Okae, H. et al. Genome-wide analysis of DNA methylation dynamics during early human development. PLoS Genet. 10, e1004868 (2014).

Sahakyan, A. et al. Human naive pluripotent stem cells model X chromosome dampening and X inactivation. Cell Stem Cell 20, 87101 (2017).

Carbognin, E., Betto, R. M., Soriano, M. E., Smith, A. G. & Martello, G. Stat3 promotes mitochondrial transcription and oxidative respiration during maintenance and induction of naive pluripotency. EMBO J. 35, 618634 (2016).

Lee, J.-H. et al. Lineage-specific differentiation is influenced by state of human pluripotency. Cell Rep. 19, 2035 (2017).

Warrier, S. et al. Direct comparison of distinct naive pluripotent states in human embryonic stem cells. Nat. Commun. 8, 15055 (2017).

Hay, D. C. et al. Efficient differentiation of hepatocytes from human embryonic stem cells exhibiting markers recapitulating liver development in vivo. Stem Cells 26, 894902 (2008).

Errichelli, L. et al. FUS affects circular RNA expression in murine embryonic stem cell-derived motor neurons. Nat. Commun. 8, 14741 (2017).

Schlaeger, T. M. et al. A comparison of non-integrating reprogramming methods. Nat. Biotechnol. 33, 5863 (2014).

Nakagawa, M. et al. Generation of induced pluripotent stem cells without Myc from mouse and human fibroblasts. Nat. Biotechnol. 26, 101106 (2007).

Wang, Y. et al. Unique molecular events during reprogramming of human somatic cells to induced pluripotent stem cells (iPSCs) at nave state. eLife 7, e29518 (2018).

Urbach, A., Bar-Nur, O., Daley, G. Q. & Benvenisty, N. Differential modeling of fragile X syndrome by human embryonic stem cells and induced pluripotent stem cells. Cell Stem Cell 6, 407411 (2010).

Blakeley, P. et al. Defining the three cell lineages of the human blastocyst by single-cell RNA-seq. Dev. Camb. Engl. 142, 31513165 (2015).

Quintanilla, R. H. Jr, Asprer, J. S. T., Vaz, C., Tanavde, V. & Lakshmipathy, U. CD44 is a negative cell surface marker for pluripotent stem cell identification during human fibroblast reprogramming. PLoS ONE 9, e85419 (2014).

Li, B. & Dewey, C. N. RSEM: accurate transcript quantification from RNA-Seq data with or without a reference genome. BMC Bioinformatics 12, 323 (2011).

Dobin, A. et al. STAR: ultrafast universal RNA-seq aligner. Bioinforma. Oxf. Engl. 29, 1521 (2013).

Robinson, M. D., McCarthy, D. J. & Smyth, G. K. edgeR: a Bioconductor package for differential expression analysis of digital gene expression data. Bioinforma. Oxf. Engl. 26, 139140 (2010).

Risso, D., Schwartz, K., Sherlock, G. & Dudoit, S. GC-content normalization for RNA-Seq data. BMC Bioinformatics 12, 480 (2011).

Krueger, F. & Andrews, S. R. Bismark: a flexible aligner and methylation caller for Bisulfite-Seq applications. Bioinforma. Oxf. Engl. 27, 15711572 (2011).

Akalin, A. et al. methylKit: a comprehensive R package for the analysis of genome-wide DNA methylation profiles. Genome Biol. 13, R87 (2012).

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Direct generation of human naive induced pluripotent stem ...

Stem Cell Glossary A Closer Look at Stem Cells

Adult stem cells A commonly used term fortissue-specific stem cells, cells that can give rise to the specialized cells in specific tissues. Includes all stem cells other than pluripotent stem cells such as embryonic and induced pluripotent stem cells.

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Autologous Cells or tissues from the same individual; an autologous bone marrow transplant involves one individual as both donor and recipient.

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Basic research Research designed to increase knowledge and understanding (as opposed to research designed with the primary goal to solve a problem).

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Blastocyst A transient, hollow ball of 150 to 200 cells formed in early embryonic development that contains the inner cell mass, from which the embryo develops, and an outer layer of cell called the trophoblast, which forms the placenta.

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Bone marrow stromal cells A general term for non-blood cells in the bone marrow, such as fibroblasts, adipocytes (fat cells) and bone- and cartilage-forming cells that provide support for blood cells. Contained within this population of cells are multipotent bone marrow stromal stem cells that can self-renew and give rise to bone, cartilage, adipocytes and fibroblasts.

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Cardiomyocytes The functional muscle cells of the heart that allow it to beat continuously and rhythmically.

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Clinical translation The process of using scientific knowledge to design, develop and apply new ways to diagnose, stop or fix what goes wrong in a particular disease or injury; the process by which basic scientific research becomes medicine.

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Clinical trial Tests on human subjects designed to evaluate the safety and/or effectiveness of new medical treatments.

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Cord blood The blood in the umbilical cord and placenta after child birth. Cord blood contains hematopoietic stem cells, also known as cord blood stem cells, which can regenerate the blood and immune system and can be used to treat some blood disorders such as leukemia or anemia. Cord blood can be stored long-term in blood banks for either public or private use. Also called umbilical cord blood.

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Cytoplasm Fluid inside a cell, but outside the nucleus.

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Differentiation The process by which cells become increasingly specialized to carry out specific functions in tissues and organs.

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Drug discovery The systematic process of discovering new drugs.

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Drug screening The process of testing large numbers of potential drug candidates for activity, function and/or toxicity in defined assays.

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Embryo Generally used to describe the stage of development between fertilization and the fetal stage; the embryonic stage ends 7-8 weeks after fertilization in humans.

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Embryonic stem cells (ESCs) Undifferentiated cells derived from the inner cell mass of the blastocyst; these cells have the potential to give rise to all cell types in the fully formed organism and undergo self-renewal.

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Fibroblast A common connective or support cell found within most tissues of the body.

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Glucose A simple sugar that cells use for energy.

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Hematopoietic Blood-forming; hematopoietic stem cells give rise to all the cell types in the blood.

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Immunomodulatory The ability to modify the immune system or an immune response.

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Induced pluripotent stem cells (iPSCs)Embryonic-like stem cells that are derived from reprogrammed, adult cells, such as skin cells. Like ESCs, iPS cells are pluripotent and can self-renew.

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In vitro Latin for in glass. In biomedical research this refers to experiments that are done outside the body in an artificial environment, such as the study of isolated cells in controlled laboratory conditions (also known as cell culture).

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In vivo Latin for within the living. In biomedical research this refers to experiments that are done in a living organism. Experiments in model systems such as mice or fruit flies are an example of in vivo research.

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Islets of Langerhans Clusters in the pancreas where insulin-producing beta cells live.

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Macula A small spot at the back of the retina, densely packed with the rods and cones that receive light, which is responsible for high-resolution central vision.

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Mesenchymal stem cells (MSCs) A term used to describe cells isolated from the connective tissue that surrounds other tissues and organs. MSCs were first isolated from the bone marrow and shown to be capable of making bone, cartilage and fat cells. MSCs are now grown from other tissues, such as fat and cord blood. Not all MSCs are the same and their characteristics depend on where in the body they come from and how they are isolated and grown. May also be called mesenchymal stromal cells.

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Multipotent stem cells Stem cells that can give rise to several different types of specialized cells in specific tissues; for example, blood stem cells can produce the different types of cells that make up the blood, but not the cells of other organs such as the liver or the brain.

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Neuron An electrically excitable cell that processes and transmits information through electrical and chemical signals in the central and peripheral nervous systems.

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Pancreatic beta cells Cells responsible for making and releasing insulin, the hormone responsible for regulating blood sugar levels. Type I diabetes occurs when these cells are attacked and destroyed by the body's immune system.

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Photoreceptors Rod or cone cells in the retina that receive light and send signals to the optic nerve, which passes along these signals to the brain.

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Placebo A pill, injection or other treatment that has no therapeutic benefit; often used as a control in clinical trials to see whether new treatments work better than no treatment.

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Placebo effect Perceived or actual improvement in symptoms that cannot be attributed to the placebo itself and therefore must be the result of the patient's (or other interested person's) belief in the treatment's effectiveness.

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Pluripotent stem cells Stem cells that can become all the cell types that are found in an embryo, fetus or adult, such as embryonic stem cells or induced pluripotent (iPS) cells.

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Preclinical research Laboratory research on cells, tissues and/or animals for the purpose of discovering new drugs or therapies.

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Precursor cells An intermediate cell type between stem cells and differentiated cells. Precursor cells have the potential to give rise to a limited number or type of specialized cells. Also called progenitor cells.

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Progenitor cells An intermediate cell type between stem cells and differentiated cells. Progenitor cells have the potential to give rise to a limited number or type of specialized cells and have a reduced capacity for self-renewal. Also called precursor cells.

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Regenerative Medicine An interdisciplinary branch of medicine with the goal of replacing, regenerating or repairing damaged tissue to restore normal function. Regenerative treatments can include cellular therapy, gene therapy and tissue engineering approaches.

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Reprogramming In the context of stem cell biology, this refers to the conversion of differentiated cells, such as fibroblasts, into embryonic-like iPS cells by artificially altering the expression of key genes.

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Retinal pigment epithelium A single-cell layer behind the rods and cones in the retina that provide support functions for the rods and cones.

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RNA Ribonucleic acid; it "reads" DNA and acts as a messenger for carrying out genetic instructions.

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Scientific method A systematic process designed to understand a specific observation through the collection of measurable, empirical evidence; emphasis on measurable and repeatable experiments and results that test a specific hypothesis.

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Self-renewal A special type of cell division in stem cells by which they make copies of themselves.

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Somatic stem cells Scientific term for tissue-specific or adult stem cells.

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Stem cells Cells that have both the capacity to self-renew (make more stem cells by cell division) and to differentiate into mature, specialized cells.

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Stem cell tourism The travel to another state, region or country specifically for the purpose of undergoing a stem cell treatment available at that location. This phrase is also used to refer to the pursuit of untested and unregulated stem cell treatments.

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TeratomaA benign tumor that usually consists of several types of tissue cells that are foreign to the tissue in which the tumor is located.

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Tissue A group of cells with a similar function or embryological origin. Tissues organize further to become organs.

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Tissue-specific stem cells Stem cells that can give rise to the specialized cells in specific tissues; blood stem cells, for example, can produce the different types of cells that make up the blood, but not the cells of other organs such as the liver or the brain. Includes all stem cells other than pluripotent stem cells such as embryonic and induced pluripotent cells. Also called adult or somatic stem cells.

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Totipotent The ability to give rise to all the cells of the body and cells that arent part of the body but support embryonic development, such as the placenta and umbilical cord.

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Translational research Research that focuses on how to use knowledge gleaned from basic research to develop new drugs, treatments or therapies.

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Zygote The single cell formed when a sperm cell fuses with an egg cell.

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Stem Cell Glossary A Closer Look at Stem Cells