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Presidential Symposium at the American Society of Gene and Cell … – PR Newswire (press release)

Jane S. Lebkowski, Ph.D., Asterias' President of R&D and Chief Scientific Officer, will be one of the presenters during the Presidential Symposium session scheduled on Friday, May 12, 2017 at 1:00pm Eastern Time. Dr. Lebkowski's presentation, titled "498 - Safety and Efficacy of Human Embryonic Stem Cell Derived Oligodendrocyte Progenitor Cells (AST-OPC1) in Patients with Subacute Cervical Spinal Cord Injury," is expected to begin at 2:15pm Eastern Time. The abstract for Dr. Lebkowski's presentation at the ASGCT meeting is available online at: http://www.abstractsonline.com/pp8/#!/4399/presentation/1996.

ASGCT is the primary professional membership organization for gene and cell therapy. The Society's members are scientists, physicians, patient advocates, and other professionals. Its members work in a wide range of settings including universities, hospitals, government agencies, foundations, biotechnology and pharmaceutical companies. Its mission is to advance knowledge, awareness, and education leading to the discovery and clinical application of gene and cell therapies to alleviate human disease.

About the SCiStar Trial

The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with sub-acute, C-5 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may retain some minimal sensory function below their injury site. AST-OPC1 is being administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.

The study is being conducted at six centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose jointly with Stanford University.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.

Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.

In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. They also received low levels of immunosuppression for the next 60 days. Delivery of AST-OPC1 was successful in all five subjects with no serious adverse events associated with AST-OPC1. No evidence of rejection of AST-OPC1 was observed in detailed immune response monitoring of all patients. In four of the five patients, serial MRI scans indicated that reduced spinal cord cavitation may have occurred. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries, which represents the first targeted population for registration trials.

About Asterias Biotherapeutics

Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.

FORWARD-LOOKING STATEMENTS

Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.

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SOURCE Asterias Biotherapeutics, Inc.

http://www.asteriasbiotherapeutics.com

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Presidential Symposium at the American Society of Gene and Cell ... - PR Newswire (press release)

Platelets suppress T cell immunity against cancer – Medical Xpress

May 5, 2017 Stylized illustration of a platelet and T cell. Plus and negative signs are used to symbolically indicate the positive (clotting) and negative (downregulating T cell immunity) effects of platelets. Credit: Emma Vought of the Medical University of South Carolina.

Blood platelets help disguise cancer from the immune system by suppressing T cells, report scientists at the Medical University of South Carolina (MUSC) in the May 5, 2017 issue of Science Immunology. In extensive preclinical tests, a promising T cell therapy more successfully boosted immunity against melanoma when common antiplatelet drugs such as aspirin were added.

Zihai Li, M.D., Ph.D., senior author on the article, is chair of the MUSC Department of Microbiology and Immunology, the program leader for the Cancer Immunology Research Program at MUSC Hollings Cancer Center, and the SmartState Sally Abney Rose Chair in Stem Cell Biology & Therapy. Li studies how tumors hide themselves from the immune system.

Li's team found that platelets release a molecule that suppresses the activity of cancer-fighting T cells. That molecule, unsurprisingly, was TGF-beta, which has been recognized for decades for its role in cancer growth.

Yet this study is the first of its kind. Most TGF-beta is inactive. Li and his group found that the surface of platelets has a protein called GARP, a molecular hook that is uniquely able to trap and activate TGF-beta. Platelets, which are small cell fragments that circulate throughout the blood and are normally involved in clotting, become the major source of activated TGF-beta that invading tumor cells use to suppress T cells. In other words, platelets help give tumors their invisibility cloak from the immune system.

Scientists have known for several years that certain cancers suppress T cells to avoid the immune system. That is why adoptive T cell therapy is one of the most promising advances in modern cancer treatment. It is a type of immunotherapy that awakens the immune system by retraining a patient's T cells to recognize their cancer. T cells are isolated from a patient's blood and retrained, or "primed," to recognize tumor cells. They are then injected back into the patient's bloodstream where they can now hunt and fight cancer.

There was some evidence that platelets might make cancer worse. For example, patients who have excessive clotting related to their cancer almost always have a worse prognosis, according to Li.

"Over the years, it has become appreciated that platelets are doing more than just clotting," says Li.

The first clue that cancer-fighting T cells might be suppressed by the body's own clotting system came when the researchers gave melanoma to mice with genetically defective platelets. Melanoma tumors grew much more slowly and primed T cells were much more active than in mice with normal platelets.

Next, the team isolated platelets and T cells from blood drawn from humans and mice. In both cases, platelets with activated clotting activity suppressed T cell response. It then used mass spectrometry to thoroughly identify the molecules released by activated platelets that most suppressed T cell activity. The molecule with the most T cell suppression was TGF-beta.

Li and his team then studied how platelets activate TGF-beta. In genetically modified mice without GARP, the molecular hook on the surface of platelets, adoptive T cell therapy was more successful at controlling melanoma. This meant that platelets without the ability to grab and activate TGF-beta were not able to suppress cancer-fighting T cells. Similar experiments confirmed this result in mice with colon carcinoma.

Finally, mice with normal platelets that were given melanoma and then adoptive T cell therapy survived longer and relapsed less when aspirin and clopidogrel, two antiplatelet drugs, were added. The researchers noted that antiplatelet drugs by themselves were not successful in combating melanoma in their experiments.

This study could inform future treatment of melanoma and other cancers and offers a sound reason to test antiplatelet drugs in clinical trials of adoptive T cell therapy. In patients with melanoma or other cancers, adoptive T cell therapy may be successful if highly available platelet-blocking drugs such as aspirin are added to the treatment. However, the current standard of care for melanoma is not adoptive T cell therapy, but so-called checkpoint inhibitors.

Li and his group want to know if combination therapy with antiplatelet drugs could improve existing cancer treatment. They are waiting for approval to begin a clinical trial that will test certain checkpoint inhibitors in combination with aspirin and clopidogrel for the treatment of patients with advanced cancers. Li's trial will complement clinical trials that are already testing adoptive T cell therapy as a single treatment for cancer.

"I'm very excited about this," says Li. "We can test simple, over-the-counter antiplatelet agents to really improve immunity and make a difference in how to treat people with cancer."

Explore further: Aspirin slows growth of colon, pancreatic tumor cells

More information: "Platelets subvert T cell immunity against cancer via GARP-TGF axis," Science Immunology (2017). immunology.sciencemag.org/lookup/doi/10.1126/sciimmunol.aai7911

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If you scanned the body for relatively higher TGF concentrations, could you use that information to find active cancers?

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Platelets suppress T cell immunity against cancer - Medical Xpress

BioNews – Unproven stem cell therapies promise versus evidence – BioNews

The ability of stem cells to divide into different mature cell types has ignited the field of regenerative medicine. Stem cells promise to repair and regenerate damaged or diseased tissues without the need for orthodox medical or surgical interventions.

However, there is disparity between the expectations held by the general public and some medical professionals versus the reality of the emerging clinical evidence. This disconnect was highlighted recently by the case of three elderly patients who were blinded by the use of an unproven stem cell therapy at a clinic in Florida, USA (BioNews 893).

While stem cell therapies in the field of haematology are showing promise, there are still many challenges in using them in any other disease models. In some countries, medical professionals are using unproven stem cell therapies as medical procedures to treat patients in lieu of conventional treatment pathways. What is more, these practitioners are operating under the premise of a trial.

Proponents of using stem cell therapies outside the context of a true clinical trial believe that these therapies are inherently safe, particularly if the stem cells derive from the individual patient themselves. And the medical professionals offering unproven stem cell therapies are convinced that the potential benefits of undergoing the therapy far outweigh the potential risks.

These benefits are used as an argument to forge ahead with unproven therapies outside ofclinical trials, as regulatory bodies are often to slow to regulate for the fast-paced field of regenerative medicine;gaining regulatory approval is usually a lengthy and costly process. And the regulation that is in place is often narrow in scope and does not account for the variety of products and manipulative techniques used in the field.

An anecdotal account of a stem cell therapys potential to cure a disease, however, does not make for an adequate standard of evidence. In the Florida case, three elderly patients with a progressive eye disease sought out an unproven stem cell therapy. The clinic involved was offering the therapy under the guise of a trial, however the patients had to pay for the procedure (in itself a 'red flag') that promised to 'cure' their disease. The therapy not only failed, but all three patients are now blind as described in The New England Journal of Medicine on 16 March.

Referring back to the disparity in expectations, how the word 'trial' is understood helps to clarify the basis of thedisconnect between the public and clinicians, and emerging evidence. The word 'trial' in the sense of a clinical trial calls for a robust experimental framework and sets of regulations and standards that safeguard the enrolled patients' rights and overall health. Furthermore, trials are performed in a phased manner to ensure any potential risks are minimised. The results generated from a certain phase informs the researchers as to the most effective way to proceed or indeed not proceed.

Using the word 'trial' in the sense of administering a therapy outside of the setting described above, however, only truly refers to the inherent risk of the therapy not working. Moreover, when being administered by a trusted medical professional, the harms of the therapy are often overshadowed by the promise of a'cure' relayed bythese professionals.

The differences in the interpretation of the word trial among medical professionals is reflected by the lack of strict regulation among professional bodies, such as medical councils and regulatory bodies including the Food & Drug Administration (FDA). While the FDA, for example, has published more specific guidelines in October 2015, these are unenforceable on a global scale.

Interestingly, on a regulatory level, there are opportunities afforded to medical professionals to use unproven stem cell therapies outside the context of a clinical trial as noted in the International Society for Stem Cell Research (ISSCR) 2016 guidelines:

...the ISSCR acknowledges that in some very limited cases, clinicians may be justified in attempting medically innovative stem cell-based interventions in a small number of seriously ill patients.

However the ISSCR goes on to clarify that it 'condemns' the use of unproven stem cell therapies in any other setting where clinical need is not deemed serious.

In relation to classifying unproven stem cell therapies as a medical procedure, the 2014 United States of America v Regenerative Sciences, LLC et al case dealt with the use of mesenchymal stem cells taken from a sample of a patient's own bone marrow to treat their own orthopaedic disorders. In this case, the court was not convinced that manipulating stem cells outside the body and reintroducing them to the patient was a matter of standard 'medical procedure', as argued by the companyRegenerative Sciences LLC. Instead, the court upheld the FDAs right to regulate the manufacturing (or manipulating) of these stem cells: however cases where there is significantly less manipulation of stem cells are yet to be tested.

Issues remain regarding how best to regulate the use of stem cell therapies, particularly in the early phases of their development. There have been calls for strict regulation through bodies such as the FDA, but others argue that strict regulation will only curtail the benefits stem cell therapies can impart. On the other hand, regulations that are too lenient, it is argued, will only harm the patient seeking the therapy, as a solid evidence base will not yet have been compiled for the therapy.

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BioNews - Unproven stem cell therapies promise versus evidence - BioNews

Scientists turn human induced pluripotent stem cells into lung cells – Medical Xpress

May 3, 2017 This is Finn Hawkins and Katherine McCauley. Credit: Jackie Ricciardi

Human lungs, like all organs, begin their existence as clumps of undifferentiated stem cells. But in a matter of months, the cells get organized. They gather together, branch and bud, some forming airways and others alveoli, the delicate sacs where our bodies exchange oxygen for carbon dioxide. The end result, ideally: two healthy, breathing lungs.

For years, scientists who study lung diseases like cystic fibrosis have tried to track this process in detail, from start to finish, in the hope that understanding how lungs form normally may help explain how things go wrong. Now, scientists at Boston University's Center for Regenerative Medicine (CReM) have announced two major findings that further our understanding of this process: the ability to grow and purify the earliest lung progenitors that emerge from human stem cells, and the ability to differentiate these cells into tiny "bronchospheres" that model cystic fibrosis. Researchers hope that the results, published separately in the Journal of Clinical Investigation and Cell Stem Cell, will lead to new, "personalized medicine" approaches to treating lung disease.

"Sorting these cells to purity is really difficult and important," says Darrell Kotton, director of CReM and co-senior author of both papers, with Brian Davis of UTHealth at the University of Texas. "It's the first step in trying to predict how an individual might respond to existing treatments or new drugs."

"There's a long list of lung diseases for which there are no treatments other than a lung transplant," added Kotton, whose work is funded by the National Institutes of Health (NIH), the Cystic Fibrosis Foundation, and the Massachusetts Life Sciences Center. "It's critically important to develop new tools for understanding these diseases."

CReM scientists work with induced pluripotent stem cells, or iPSCs, which were discovered by Shinya Yamanaka in 2006. Yamanaka figured out how to take an adult cell in the human bodylike a blood cell or skin celland "reprogram" it into a stem cell with the ability to grow into any organ. In recent years, several groups of scientists have grown lung cells from human iPSCs, but the recipes aren't perfectthe resulting lung cells grow amidst a jumble of liver cells, intestinal cells, and other tissues.

"That's a big issue," says Finn Hawkins, a BU School of Medicine (MED) assistant professor of medicine and part of the CReM team. Hawkins is co-first author on the Journal of Clinical Investigation paper, along with Philipp Kramer, formerly of UTHealth. "If you want to use these cells to study the lung, you need to get rid of those others."

First, Hawkins needed a way to identify the lung cells. Previous work by Kotton and other CReM scientists demonstrated that mouse stem cells express a gene called Nkx2-1 at the "fate decision"the moment they turn into lung cells. "That's the first gene that comes on that says, 'I'm a lung cell,'" says Hawkins. Kotton built a reporter gene that glowed green when the stem cells first expressed Nkx2-1, and Hawkins engineered the same gene into human cells. Now, he could easily spot and purify the glowing green lung cells.

Using a flow cytometer, Hawkins and his colleagues separated the green cells out from the mix, then grew them in a matrix. The result: tiny green spheres about half a millimeter across, "a population of pure, early lung cells," says Hawkins. The team calls the tiny spheres "organoids," simplified and miniaturized versions of an organ, containing key types of lung cells. The organoids are tools, and they serve at least two important purposes. First, they allow scientists to study, in detail, a critical juncture in human lung development about which very little is known. "We discovered that many of the genes that control lung development in other species, such as mice, are also expressed in these human cells," says Hawkins.

The organoids serve another purpose, as well: scientists can grow them into more mature, specific cell typeslike airway cells or alveolar cellsthat are critical for lung function. "Now we can actually start looking at disease," says Hawkins. That's where Katherine McCauley (MED'17), a fifth-year PhD candidate at CReM, enters the picture.

McCauley's interest is cystic fibrosis, a disease caused by mutations in a single gene, CFTR. The mutation causes a person's lungs to produce a thick, viscous mucus that leads to infection, inflammation, and, eventually, lung failure. For many patients, there is no cure.

McCauley, looking at the earliest stages of the disease, wanted to take Hawkins' purified lung cells to the next step and figure out how they became airway cells. Through many painstaking experiments, she zeroed in on a signaling pathway called Wnt, known to be important in mouse lung development. By turning the pathway off, she guided the immature lung cells into becoming airway cells. Then, she grew them into tiny balls of cells, which she called "bronchospheres."

Like Hawkins' organoids, the bronchospheres don't act like a bronchus; they are simply a collection of specific cells. But their specificity makes them exquisitely useful. "We wanted to see if we could use these to study airway diseases," says McCauley. "That's one of the big goals: to engineer these cells from patients and then use them to study those patients' diseases."

As a proof of concept, McCauley obtained two cell lines from a patient with cystic fibrosis, one in which the CFTR mutation that caused the disease had been corrected, and one in which it hadn't, and grew them into bronchospheres. To see if her recipe worked, she ran a test, applying a drug that should cause spheres made of normal, functioning cells to fill with fluid. It worked: the "fixed" bronchospheres began to swell, while the cystic fibrosis spheres didn't react. "The cool part is that we measured this using high-throughput microscopy, and then we calculated the change in area with time," says McCauley, who published these results in Cell Stem Cell and is lead author on the study. "So now we can evaluate CFTR function in a quantitative way."

The next step, says McCauley, is to improve the test, and scale it up, and create similar tests for other lung diseases. "The end goal is to take cells from a patient, and then screen different combinations of drugs," she says. "The idea that we could take a patient's cells and test not twenty, but hundreds or thousands of drugs, and actually understand how the patient was going to respond before we even give them the treatment, is just an incredible idea."

Explore further: New study makes strides towards generating lung tissue

Using Induced pluripotent stem cells (iPSCs), researchers have for the first time profiled the complete genetic programs of early lung progenitors identifying genes that control lung formation and have created mini-lung organoids ...

Researchers have developed a new approach for growing and studying cells they hope one day will lead to curing lung diseases such as cystic fibrosis through "personalized medicine."

Researchers have discovered a new efficient way to generate thyroid cells, known as thyrocytes, using genetically modified embryonic stem cells.

A new study has found that stem cell therapy can reduce lung inflammation in an animal model of chronic obstructive pulmonary disease (COPD) and cystic fibrosis. Although, still at a pre-clinical stage, these findings have ...

Researchers at Boston University School of Medicine (BUSM) and Boston Medical Center (BMC) have derived a population of pure lung and thyroid progenitor cells in vitro that successfully mimic the developmental milestones ...

Researchers at the University of Toronto and the Hospital for Sick Children (SickKids) are paving the way towards individualized medicine for patients with cystic fibrosis.

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Scientists turn human induced pluripotent stem cells into lung cells - Medical Xpress

Recent Study Published in Nature Unravels a Novel Pathway for … – Business Wire (press release)

PLEASANTON, Calif.--(BUSINESS WIRE)--10x Genomics, a company focused on enabling the mastery of biology by accelerating genomic discovery, today announced publication of an article in the journal Nature of a collaborative research study with researchers at the Stanford University School of Medicine. The article entitled, Non-equivalence of Wnt and R-spondin ligands during Lgr5+ intestinal stem-cell self-renewal, utilizes the 10x Genomics Single Cell 3 Solution for single-cell RNA-seq (scRNA-seq) to unravel the priming and self-renewal mechanisms of intestinal stem cells (ISCs).

The renewal and differentiation of Lgr5+ ISCs is critical to the continuous regeneration of the epithelial lining of the gut, which enables us to absorb nutrients and provides a barrier to protect us from the external environment. Disruptions in this process can lead to or worsen human intestinal disorders such as inflammatory bowel disease (IBD), gastrointestinal cancer and Celiac disease.

This carefully regulated process occurs within a stem-cell niche called the intestinal crypt, and depends on Wnt signaling, which can be turned up by Wnt and R-spondin (RSPO) ligands. The authors sought to identify the unique functional roles of Wnt and RSPO ligands for regulating Lgr5+ ISCs and the relative contributions of both ligands to in vivo Wnt signaling and stem-cell biology.

The authors were able to show using in vivo experiments that Wnt and RSPO are not redundant signals. RSPO was shown to expand stem cell number. Although Wnt was needed to maintain Lgr5+ ISCs in the presence of RSPO, Wnt was not sufficient to induce additional numbers of Lgr5+ ISCs above a certain threshold, demonstrating that RSPO, and not Wnt, establishes the set point for Lgr5+ ISC number. The authors performed single-cell RNA-seq to definitively show that the signaling contributions of Wnt and RSPO elicited distinct effects on ISCs, by fully characterizing the expression profile for each unique cellular subtype on a cell-by-cell basis upon perturbation of those signals in vivo.

By characterizing gene expression from 13,102 single cells, Yan and colleagues were able to show that Lgr5- control cells represented differentiated cell types of the small intestinal lineages, including Paneth, goblet, enteroendocrine, enterocyte, pre-enterocyte, and tuft cells. The Lgr5+ cells consisted of three cellular sub-populations, corresponding to cycling stem cells, non-cycling stem cells, and transit amplifying cells. The authors were able to further show that these three distinct sub-populations of Lgr5+ cells were each uniquely affected by perturbations in Wnt and RSPO signaling, conclusively demonstrating that Wnts are priming factors that enable stem cells to be competent by expressing RSPO receptors on their cell surface, whereas RSPOs are actual self-renewal factors that expand stem cell number.

Single-cell analysis provided conclusive evidence for the unique roles of Wnt and RSPO signaling to their respective function, either co-operatively priming Lgr5+ cells for competency, or for RSPO-mediated self-renewal. said Grace Zheng, Ph.D., research scientist at 10x Genomics. This powerfully illustrates the utility of single-cell RNA-seq to monitor discrete stem-cell states and their dynamic perturbation. To do this with any other technology would have been extremely cumbersome, if not impossible.

We are very excited about this result, and it opens up the possibility that analogous multi-tiered regulation by priming and self-renewal factors may be a generalized property of stem cells across other organ systems, either through Wnt and RSPO or functionally equivalent stem-cell niche components, said Ben Hindson, Ph.D., president, co-founder, and chief scientific officer of 10x Genomics. This could have wide reaching implications for stem-cell research and potentially yield new insight towards therapeutic applications in the future.

The lead author of the study is Kelley Yan, M.D. Ph.D., lead author of the study, formerly a postdoctoral fellow at Stanford and now an Assistant Professor of Medicine and of Genetics and Development in the Columbia Center for Human Development at Columbia University Medical Center. The senior author is Calvin Kuo, M.D. Ph.D., Professor of Medicine at Stanford.

The article entitled, Non-equivalence of Wnt and R-spondin ligands during Lgr5+ intestinal stem-cell self-renewal, can be accessed from Nature online: http://dx.doi.org/10.1038/nature22313

For more information about this research, visit the Kuo Lab at Stanford: http://kuolab.stanford.edu/index.html

About 10x Genomics

10x Genomics is changing the definition of sequencing by providing an innovative genomics platform that dramatically upgrades the capabilities of existing sequencing technologies. This is achieved through a combination of new microfluidic science, chemistry and bioinformatics. By implementing GemCode Technology within the Chromium System, researchers can now, for the first time, find new structural variants, haplotypes and other valuable genomic information with comprehensive workflows for Single Cell, V(D)J, Genome, Exome and de novo Assembly applications that incorporate their pre-existing sequencing technologies. http://www.10xGenomics.com.

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Recent Study Published in Nature Unravels a Novel Pathway for ... - Business Wire (press release)

Breakthrough Regenerative Therapeutics Company Establishes Scientific Advisory Board – Yahoo Finance

MONTREAL, May 4, 2017 /PRNewswire/ --Fortuna Fix Inc.("Fortuna"), a private, clinical-stage biotech company, is aiming to be the first to eliminate the need for embryonic and fetal stem cells by using direct reprogramming of autologous cells to treat neurodegenerative diseases. Fortuna announced today the launch of its Scientific Advisory Board ("SAB") with Professor Michael Fehlings, MD, PhD; Father Kevin FitzGerald, S.J., PhD; Col. (R) Dallas Hack, MD, MPH; and Professor James Giordano, PhD.

"We are excited and honored to have these world-leading experts join our SAB," says CEO Jan-Eric Ahlfors. "We look forward to working with them to bring our novel regenerative medicine solutions to patients suffering from neurotrauma and neurodegeneration."

Fortuna's two flagship technologies autologous directly reprogrammed neural precursor cells ("drNPC") and Regeneration Matrix ("RMx") are poised to lead a revolution in neuro-regeneration.

For the first time, patients suffering from neurotrauma or neurodegeneration will be able to get treated with autologous neural stem cells produced by direct reprogramming (i.e. starting with and only using the patient's own cells, bypassing use of pluripotent stem cells and avoiding harvesting and use of human embryos or fetuses). The method of direct reprogramming developed by Fortuna relies on an ethical, rapid, high throughput, low cost and fully automated manufacturing process. As drNPC do not involve any genetic engineering, pluripotent stem cells, or use of immune-suppression, it provides patients with personalized stem cells that are also expected to have a greater safety profile. In addition, drNPC are expected to replace dead neural cells, something that no other current technology can do effectively.

RMx is a unique and highly efficient bio-scaffold for the promotion of neural tissue regrowth.

"Our testing of drNPC at the Krembil Neuroscience Centre of the University Health Network in various Spinal Cord Injury ("SCI") animal models to characterize their regenerative capacity and safety profile indicates that drNPC are a promising source of therapeutic stem cells with potential for tissue preservation and functional improvement after SCI. I am highly encouraged by the reprogramming efficiency of drNPC and look forward to leading the clinical development of drNPC for SCI," says Professor Fehlings, after working on the drNPC in his lab for two years.

Dr. Hack further remarks:"Fortuna's autologous drNPC represent a major advance in cell therapy for treatment of CNS injury and degeneration. For the first time, neurons, astrocytes and oligodendrocytes the three type of cells of the brain and spinal cord can be repaired and replaced where these cells have died or been destroyed due to trauma or neurodegenerative disease. Fortuna's proprietary automated manufacturing addresses a key hurdle of personalized cell therapy, making drNPC commercially viable both at small and large scale"

"Stem cell therapeutics have been plagued with controversy and hype, raising ethical and political issues that have resulted in a relatively hostile funding environment for research and development in the field. I am excited to work alongside Fortuna to help advance development of their ethical and commercially viable platform for cell therapeutics to benefit patients, their families, and our entire society," says Father FitzGerald.

The SAB members encompass unique expertise in key areas of importance for the company:

Professor Michael G. Fehlings, MD, PhD, FRCSC, FACS

Dr. Michael Fehlings is a world-renowned Neurosurgeon focusing on Spinal Cord Injury and a leader in the field of stem cell therapeutics for SCI. Dr. Fehlings is the Vice Chair of Research for the Department of Surgery, Co-Director of the Spine Program and a Professor of Neurosurgery at the University of Toronto. He is well known for his work on early decompressive surgery, which demonstrated significant improvement on neurological and functional outcomes after SCI that had an important impact on how spinal trauma is managed today. Recently, during the Henry Farfan Award ceremony (2013), he was described as the "single most influential active spinal cord injury researcher and clinician in the world."Dr. Fehlings is also the recipient of the coveted Olivecrona Award from the Karolinska Institute in Stockholm, Sweden (known as the "Nobel Prize of Neuroscience").

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Dr. Fehlings has been an integral part of the work performed by independent validators sponsored by CIHR (Canadian Institutes of Health Research) on Fortuna's technology. Dr. Fehlings' work was presented at the annual International Society for Stem Cell Research (ISSCR) conference in June 2016 in San Francisco with a follow up to be presented at the ISSCR in June 2017 in Boston.

Father Kevin T. FitzGerald, S.J., PhD, PhD

Father Kevin FitzGerald is a Professor at Georgetown University and advisor to the Vatican on Bioethics (including human genetic engineering, cloning, stem cell research, and personalized medicine). Father FitzGerald is the Dr. David Lauler Chair of Catholic Health Care Ethics in the Center for Clinical Bioethics at Georgetown University, and an Associate Professor in the Department of Oncology at the Georgetown University Medical Center. He is a founding member of Do No Harm, a member of the ethics committee for the March of Dimes, a member of the Genetic Alliance IRB, and a member of the Georgetown-MedStar Hospital Ethics Committee. Father FitzGerald has been a Corresponding Member of the Pontifical Academy of Life since 2005, and has been a Consultor to the Pontifical Council for Culture since 2014. He has a Ph.D. in molecular genetics, and a second Ph.D. in bioethics, from Georgetown University. His research efforts focus on the investigation of abnormal gene expression in cancer, and on ethical issues in biomedical research and medical genomics.

Col. (R) Dallas Hack, MD, MPH, MMS, CPE

Dr. Dallas Hack, recently retired from the US military, is one of the leaders of military medicine of his time, with a particular focus on brain health (Traumatic Brain Injury ("TBI") and concussion). He served as the Director of the US Army Combat Casualty Care Research Program and Chair of the Joint Program Committee for Combat Casualty Care from 2008 to 2014 and as the Senior Medical Advisor to the Principal Assistant for Research and Technology, US Army Medical Research and Materiel Command from 2014 to 2015. He coordinated more than 70% of the Department of Defense trauma research to improve battlefield trauma care of those injured in combat at a time when the Department of Defense funded more TBI research than any other organization in the world because of the increasing awareness of the massive burden of TBI in the military. He has held numerous military medical leadership positions, including Chief of Clinical Services at Fort Knox, KY, Commander of the NATO Headquarters Healthcare Facility, and Command Surgeon at the strategic level during Operations Enduring Freedom and Iraqi Freedom.

Col. (R) Dallas Hack has received numerous military awards, including the Bronze Star, two Legion of Merit awards, and seven Meritorious Service Medals and was inducted as a Distinguished Member of the Military Order of Medical Merit. He has appointments from the School of Medicine, University of Pittsburgh as Adjunct Professor of Neurosurgery, and from the Department of Physical Medicine and Rehabilitation, School of Medicine, Virginia Commonwealth University as Associate Clinical Professor.

Professor James Giordano, PhD, MPhil

Dr. James Giordano is Professor in the Departments of Neurology and Biochemistry, and Chief of the Neuroethics Studies Program at the Pellegrino Center for Clinical Bioethics of Georgetown University Medical Center, Washington, DC. Prof.Giordano has served as a member of the Neuroethics, Legal and Social Issues Advisory Panel of the Defense Advanced Research Projects' Agency (DARPA), as a Senior Science Advisory Fellow of the Strategic Multilayer Assessment Branch of the Joint Staff of the Pentagon, is an appointed member of the Secretary of Health and Human Services Advisory Council for Human Research Protection, and is a Research Fellow of the European Union Human Brain Project. In recognition of his ongoing work, Prof. Giordano was elected to the European Academy of Science and Arts.

About Fortuna Fix Inc.

Fortuna is a private, clinical-stage biotech company with a patented direct cell reprogramming technology platform together with a patented bio-scaffolding technology for treatment of neurodegenerative diseases and neurotrauma. The company is focused on clinical development of its platforms for a range of neurodegenerative diseases including SCI, Parkinson's disease, stroke, TBI, and ALS. The company has developed a proprietary fully automated GMP manufacturing system for production of drNPC, initially to be used in clinical trials in Parkinson's disease and Spinal Cord Injury.

Media contact

Vikram Lamba, CFO Email: vikramlamba09@gmail.com http://www.fortunafix.com

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Breakthrough Regenerative Therapeutics Company Establishes Scientific Advisory Board - Yahoo Finance

Human Embryonic Stem Cells (HESC) Market by 2022 -Growth … – MilTech

Human Embryonic Stem Cells (HESC) Market is expected to witness growth of international market with respect to advancements and innovations including development history, competitive analysis and regional development forecast.

The report starts with a basic Human Embryonic Stem Cells (HESC) market overview. In this introductory section, the research report incorporates analysis of definitions, classifications, applications and industry chain structure.

In depth analysis of Human Embryonic Stem Cells (HESC) Marketis a crucial thing for various stakeholders like investors, CEOs, traders, suppliers and others.

Human Embryonic Stem Cells (HESC) Market split by product type,with production, revenue, price, market share and growth rate of each type, can be divided into

Adult Sources

Fetal Sources

Human Embryonic Stem Cells (HESC) Market split by application,report focuses on consumption, market share and growth rate of Human Embryonic Stem Cells (HESC) in each application and can be divided into

Regenerative Medicine

Stem Cell Biology Research

Tissue Engineering

Toxicology Testing

Browse more detail information about Human Embryonic Stem Cells (HESC) Market at:http://www.360marketupdates.com/10570506

To begin with, the report elaborates the Human Embryonic Stem Cells (HESC) Market overview. Various definitions and classification of the industry, applications of the industry and chain structure are given. Present day status of the Human Embryonic Stem Cells (HESC) Market in key regions is stated and industry policies and news are analysed.

Following are the key players covered in this Human Embryonic Stem Cells (HESC) Market research report:

Astellas Pharma Inc/ Ocata Therapeutics

STEMCELL Technologies

BIOTIME, INC

Thermo Fisher Scientific

CellGenix

ESI BIO

PromoCell

Lonza

Kite Pharma

Cynata

Sumanas

LifeCell

And Many Others

Get a PDF Sample of Human Embryonic Stem Cells (HESC) Market Research Report at:http://www.360marketupdates.com/enquiry/request-sample/10570506

After the basic information, the Human Embryonic Stem Cells (HESC) Market report sheds light on the production. Production plants, their capacities, global production and revenue are studied. Also, the Human Embryonic Stem Cells (HESC) Market growth in various regions and R&D status are also covered.

Following are Major Table of Content of Human Embryonic Stem Cells (HESC) Industry:

Human Embryonic Stem Cells (HESC) Market Competition by Manufacturers

Human Embryonic Stem Cells (HESC) Production, Revenue (Value) by Region (2017-2022)

Human Embryonic Stem Cells (HESC) Supply (Production), Consumption, Export, Import by Regions (2017-2022)

Human Embryonic Stem Cells (HESC) Production, Revenue (Value), Price Trend by Type

Human Embryonic Stem Cells (HESC) Market Analysis by Application

Human Embryonic Stem Cells (HESC) Manufacturers Profiles/Analysis

Human Embryonic Stem Cells (HESC) Manufacturing Cost Analysis

Industrial Chain, Sourcing Strategy and Downstream Buyers

Further in the Human Embryonic Stem Cells (HESC) Market Industry Analysis report, the Human Embryonic Stem Cells (HESC) Market is examined for price, cost and gross capacity. These three points are analysed for types, companies and regions. In continuation with this data sale price for various types, applications and region is also included. The Human Embryonic Stem Cells (HESC) Market for major regions is given.

Scope of the Human Embryonic Stem Cells (HESC) Industry on the basis of region:

The West

Southwest

The Middle Atlantic

New England

The South

The Midwest

Additionally, type wise and application wise consumption figures are also given. With the help of supply and consumption data, gap between these two is also explained.

To provide information on competitive landscape, this report includes detailed profiles of Human Embryonic Stem Cells (HESC) Market key players. For each player, product details, capacity, price, cost, gross and revenue numbers are given. Their contact information is provided for better understanding.

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Human Embryonic Stem Cells (HESC) Market by 2022 -Growth ... - MilTech

HIV breakthrough: Scientists remove virus in animals using gene editing – Medical News Today

Worldwide, tens of millions of people are living with HIV. While scientists and medical professionals do not yet have a permanent cure for the virus, researchers have just made a breakthrough: they managed to eliminate the HIV-1 infection in mice.

According to the Centers for Disease Control and Prevention (CDC), more than 36 million people across the world are HIV positive, and approximately 1.2 million people in the United States live with the virus.

While there is currently no cure for the infection, scientists have just moved closer to finding one. Using a gene editing technology called "CRISPR/Cas9," the researchers successfully excised the HIV-1 provirus in three animal models.

A provirus is an inactive form of virus. It occurs when the virus has integrated into the genes of a cell. In the case of HIV, these host cells are the so-called CD4 cells - once the virus has been incorporated into the DNA of the CD4 cells, it replicates itself with each generation of CD4 cells.

The three mouse models used in the current research included a "humanized" model, in which the mice were genetically modified to have human immune cells, which were then infected with HIV-1.

The team was co-led by Dr. Wenhui Hu, Ph.D., associate professor in the Center for Metabolic Disease Research and the Department of Pathology at the Lewis Katz School of Medicine (LKSOM) at Temple University in Philadelphia, together with Kamel Khalili, Ph.D., Laura H. Carnell Professor and chair of the Department of Neuroscience at LKSOM, and Won-Bin Young, Ph.D, who just recently joined LKSOM.

The new study - published in the journal Molecular Therapy - builds on previous research by the same team, during which they used genetically modified rodents to demonstrate that their gene editing technology could eliminate the HIV-1-infected segments of DNA.

"Our new study is more comprehensive," Dr. Hu explains. "We confirmed the data from our previous work and have improved the efficiency of our gene editing strategy. We also show that the strategy is effective in two additional mouse models, one representing acute infection in mouse cells and the other representing chronic, or latent, infection in human cells."

Dr. Hu and team inactivated HIV-1, significantly reducing the RNA expression of viral genes in the organs and tissues of genetically modified mice.

Specifically, the RNA expression was reduced by approximately 60 to 95 percent.

The researchers then tested their findings by acutely infecting mice with EcoHIV - the equivalent of the HIV-1 in humans. Dr. Khalili explains the procedure:

"During acute infection, HIV actively replicates. With EcoHIV mice, we were able to investigate the ability of the CRISPR/Cas9 strategy to block viral replication and potentially prevent systemic infection."

The CRISPR/Cas9 method was up to 96 percent efficacious in eradicating EcoHIV in mice.

Finally, in the third model, mice received a transplant of human immune cells, including T cells, which were then infected with HIV-1.

One of the main reasons that a cure for HIV has yet to be discovered is the virus's ability to "hide" in the genomes of T cells, where it lives latently. This is why researchers applied the CRISPR/Cas9 technology to these mice with infected T cells.

After a single round of gene editing, the viral segments were excised from the human cells that had been integrated into the mouse tissues and organs. They removed the provirus from the mice's spleen, lungs, heart, colon, and brain after only one therapy injection.

The injection was with "quadruplex sgRNAs/saCas9 AAV-DJ/8" - an improved adeno-associated viral (AAV) vector.

AAV vectors are commonly used in gene therapy, but "the AAV-DJ/8 subtype combines multiple serotypes, giving us a broader range of cell targets for the delivery of our CRISPR/Cas9 system," Dr. Hu explains.

To assess the success of the genetic interventions, the team measured HIV-1 RNA levels using live bioluminescence imaging.

This is the first time that a team of researchers has managed to halt the replication of the HIV-1 virus and eliminate it completely from the infected cells in animals.

The team also provided the first evidence that HIV-1 can be successfully eradicated and full infection with the virus can be prevented using the CRISPR/Cas9 gene editing strategy.

The study was deemed "a significant step towards human clinical trials" by the authors, and the findings represent a breakthrough in the search for an HIV cure.

"The next stage would be to repeat the study in primates, a more suitable animal model where HIV infection induces disease, in order to further demonstrate elimination of HIV-1 DNA in latently infected T cells and other sanctuary sites for HIV-1, including brain cells. Our eventual goal is a clinical trial in human patients."

Kamel Khalili, Ph.D.

Learn how an HIV 'fingerprint' tool could greatly assist vaccine development.

Follow this link:
HIV breakthrough: Scientists remove virus in animals using gene editing - Medical News Today

Cellaria and Biological Industries USA Partner on Stem Cell Media … – EconoTimes

Thursday, May 4, 2017 11:31 AM UTC

CAMBRIDGE, Mass. and CROMWELL, Conn., May 04, 2017 -- Cellaria, LLC, a scientific innovator that develops revolutionary new patient-specific models for challenging diseases, and Biological Industries USA (BI-USA), a subsidiary of Biological Industries (Israel), today announced a new sales and marketing agreement to promote custom stem cell services. The partnership combines BI-USAs strength in stem cell culture media and manufacturing with Cellarias comprehensive Stem Cell Services program, which includes industry leading RNA reprogramming and custom differentiation services. Together, the companies will offer one of the industrys most innovative and comprehensive stem cell service offerings available to biotechnology companies and academic institutions.

As part of the agreement, Cellaria will distribute BI-USAs stem cell media offering, including its NutriStem hPSC Medium, a cGMP xeno-free media specifically designed for human pluripotent stem cell culture. Cellaria will also incorporate the product into its stem cell services. BI-USA will market Cellaria's customized stem cell services, establishing an integrated, single source solution for iPS cell line derivation, culture maintenance, banking, characterization and differentiation services.

BI is one of the most respected names in life sciences today, said David Deems, chief executive officer at Cellaria. The companys strong market presence and innovative media products will enhance our stem cell and RNA reprogramming service offerings and significantly increase the availability and appeal of our combined offerings.

This is an important partnership for us, added Tanya Potcova, chief executive officer of BI-USA. In combination, our teams bring a wealth of stem cell experience but also share a common goal of creating higher quality, more consistent research outcomes for researchers in the life sciences field. We are pleased to be working with the team at Cellaria to put the best possible tools and support in the hands of our present and future customers.

Please visit Cellaria and BI at the International Society of Stem Cell Research Annual Meeting in Boston, MA June 14-17, 2017 at booth# 407.

About Cellaria Cellaria creates high quality, next generation in vitro disease models that reflect the unique nature of a patients biology. All models begin with tissue from a patient, capturing clinically relevant details that inform model characterization. For cancer, Cellarias cell models exhibit molecular and phenotypic characteristics that are highly concordant to the patient. For RNA-mediated iPS cell line derivation and stem cell services, Cellarias cell models enable interrogation of patient and disease-specific mechanisms of action. Cellarias innovative products and services help lead the research community to more personalized therapeutics, revolutionizing and accelerating the search for a cure. For more information, visitwww.cellariabio.com.

About Biological Industries Biological Industries (BI) is one of the worlds leading and trusted suppliers to the life sciences industry, with over 35 years experience in cell culture media development and cGMP manufacturing. BIs products range from classical cell culture media to supplements and reagents for stem cell research and potential cell therapy applications, to serum-free, xeno-free media. BI is committed to a Culture of Excellence through advanced manufacturing and quality-control systems, regulatory expertise, in-depth market knowledge, and extensive technical customer-support, training, and R&D capabilities.

Biological Industries USA (BI-USA) is the US commercialization arm of BI, with facilities in Cromwell, Connecticut. Members of the BI-USA team share a history and expertise of innovation and success in the development of leading-edge technologies in stem cell research, cellular reprogramming, and regenerative medicine. For more information, visit http://www.bioind.com or connect onLinkedIn,Twitter, andFacebook.

Human Life Could Be Extended Indefinitely, Study Suggests

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Could a contraceptive app be as good as the pill?

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Society actually does want policies that benefit future generations

Six cosmic catastrophes that could wipe out life on Earth

Big Pharma Starts Using Cannabis For Making Drugs In Earnest

Do you need to worry if your baby has a flat head?

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Cellaria and Biological Industries USA Partner on Stem Cell Media ... - EconoTimes

Phys.org – embryonic stem cells

The formation of a human embryo starts with the fertilization of the oocyte by the sperm cell. This yields the zygote, the primordial cell that carries one copy each of the maternal and paternal genomes. However, this genetic ...

Scientists are getting closer to understanding how naked mole rats, the world's longest living rodent species, avoid cancer, which could lead to safer stem cell therapies for human diseases.

Researchers at Karolinska Institutet have identified cell surface markers specific for the very earliest stem cells in the human embryo. These cells are thought to possess great potential for replacing damaged tissue but ...

Scientists have determined the first 3D structures of intact mammalian genomes from individual cells, showing how the DNA from all the chromosomes intricately folds to fit together inside the cell nuclei.

University of Tsukuba-led researchers explored the function of the reprogramming factor KLF4 in production of induced pluripotent stem cells (iPSCs). KLF4 was shown to bind upstream of the Tcl1 target gene, which controls ...

Scientists at the University of Cambridge have managed to create a structure resembling a mouse embryo in culture, using two types of stem cells - the body's 'master cells' - and a 3D scaffold on which they can grow.

An International Reserach Team coordinated by Igb-Cnr has discovered a key role of vitamins and amino acids in pluripotent stem cells. The research is published in Stem Cell Reports, and may provide new insights in cancer ...

A new nanofiber-on-microfiber matrix could help produce more and better quality stem cells for disease treatment and regenerative therapies.

A new report from the Stowers Institute for Medical Research chronicles the embryonic origins of planaria, providing new insight into the animal's remarkable regenerative abilities.

Freiburg plant biologist Prof. Dr. Thomas Laux and his research group have published an article in the journal Developmental Cell presenting initial findings on how shoot stem cells in plants form during embryogenesis, the ...

Embryonic stem cells (ES cells) are stem cells derived from the inner cell mass of an early stage embryo known as a blastocyst. Human embryos reach the blastocyst stage 45 days post fertilization, at which time they consist of 50150 cells.

Embryonic Stem (ES) cells are pluripotent. This means they are able to differentiate into all derivatives of the three primary germ layers: ectoderm, endoderm, and mesoderm. These include each of the more than 220 cell types in the adult body. Pluripotency distinguishes ES cells from multipotent progenitor cells found in the adult; these only form a limited number of cell types. When given no stimuli for differentiation, (i.e. when grown in vitro), ES cells maintain pluripotency through multiple cell divisions. The presence of pluripotent adult stem cells remains a subject of scientific debate; however, research has demonstrated that pluripotent stem cells can be directly generated from adult fibroblast cultures.

Because of their plasticity and potentially unlimited capacity for self-renewal, ES cell therapies have been proposed for regenerative medicine and tissue replacement after injury or disease. However Diseases treated by these non-embryonic stem cells include a number of blood and immune-system related genetic diseases, cancers, and disorders; juvenile diabetes; Parkinson's; blindness and spinal cord injuries. Besides the ethical concerns of stem cell therapy (see stem cell controversy), there is a technical problem of graft-versus-host disease associated with allogeneic stem cell transplantation. However, these problems associated with histocompatibility may be solved using autologous donor adult stem cells or via therapeutic cloning.

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Phys.org - embryonic stem cells