Shinya Yamanaka ( , Yamanaka Shin'ya?, born September 4, 1962) is a Japanese Nobel Prize-winning stem cell researcher.[1][2][3] He serves as the director of Center for iPS Cell Research and Application and a professor at the Institute for Frontier Medical Sciences at Kyoto University; as a senior investigator at the UCSF-affiliated J. David Gladstone Institutes in San Francisco, California; and as a professor of anatomy at University of California, San Francisco (UCSF). Yamanaka is also a past president of the International Society for Stem Cell Research (ISSCR).

He received the Wolf Prize in Medicine in 2011 with Rudolf Jaenisch;[6] the Millennium Technology Prize in 2012 together with Linus Torvalds. In 2012 he and John Gurdon were awarded the Nobel Prize for Physiology or Medicine for the discovery that mature cells can be converted to stem cells.[7] In 2013 he was awarded the $3 million Breakthrough Prize in Life Sciences for his work.

Yamanaka was born in Higashisaka Japan in 1962. After graduating from Tennji High School attached to Osaka Kyoiku University,[8] he received his M.D. at Kobe University in 1987 and his PhD at Osaka City University Graduate School in 1993. After this, he went through a residency in orthopedic surgery at National Osaka Hospital and a postdoctoral fellowship at the Gladstone Institute of Cardiovascular Disease, San Francisco.

Afterwards he worked at the Gladstone Institutes in San Francisco, USA and Nara Institute of Science and Technology in Japan. Yamanaka is currently Professor at Kyoto University, where he directs its Center for iPS Research and Application. He is also a senior investigator at the Gladstone Institutes as well as the director of the Center for iPS Cell Research and Application.[9]

Between 1987 and 1989, Yamanaka was a resident in orthopedic surgery at the National Osaka Hospital. His first operation, was removing a benign tumor from his friend Shuichi Hirata, a task he could not complete after one hour, when a skilled surgeon would take ten minutes or so. Some seniors referred to him as "Jamanaka", a pun on the Japanese word for obstacle.[10]

From 1993 to 1996, he was at the Gladstone Institute of Cardiovascular Disease. Between 1996 and 1999, he was an assistant professor at Osaka City University Medical School, but found himself mostly looking after mice in the laboratory, not doing actual research.[10]

His wife advised him to become a practicing doctor, but instead he applied for a position at the Nara Institute of Science and Technology. He stated that he could and would clarify the characteristics of embryonic stem cells, and this can-do attitude won him the job. From 19992003, he was an associate professor there, and started the research that would later win him the 2012 Nobel Prize. He became a full professor and remained at the institute in that position from 20032005. Between 2004 and 2010, Yamanaka was a professor at the Institute for Frontier Medical Sciences.[11] Currently, Yamanaka is the director and a professor at the Center for iPS Cell Research and Application at Kyoto University.

In 2006, he and his team generated induced pluripotent stem cells (iPS cells) from adult mouse fibroblasts.[1] iPS cells closely resemble embryonic stem cells, the in vitro equivalent of the part of the blastocyst (the embryo a few days after fertilization) which grows to become the embryo proper. They could show that his iPS cells were pluripotent, i.e. capable of generating all cell lineages of the body. Later he and his team generated iPS cells from human adult fibroblasts,[2] again as the first group to do so. A key difference from previous attempts by the field was his team's use of multiple transcription factors, instead of transfecting one transcription factor per experiment. They started with 24 transcription factors known to be important in the early embryo, but could in the end reduce it to 4 transcription factors Sox2, Oct4, Klf4 and c-Myc.[1]

Yamanaka practiced judo (2dan black belt) and played rugby as a university student. He also has a history of running marathons. After a 20-year gap, he competed in the inaugural Osaka Marathon in 2011 as a charity runner with a time of 4:29:53. He also took part in the 2012 Kyoto Marathon to raise money for iPS research, finishing in 4:03:19. He also ran in the second Osaka Marathon on November 25, 2012.[12]

In 2007, Yamanaka was recognized as a "Person Who Mattered" in the Time Person of the Year edition of Time Magazine.[13] Yamanaka was also nominated as a 2008 Time 100 Finalist.[14] In June 2010, Yamanaka was awarded the Kyoto Prize for reprogramming adult skin cells to pluripotential precursors. Yamanaka developed the method as an alternative to embryonic stem cells, thus circumventing an approach in which embryos would be destroyed.

See more here:
Shinya Yamanaka - Wikipedia, the free encyclopedia

12 hours ago Fruit fly larva are used to study stem cells key features. Credit: Wikipedia

The study, performed with fruit flies, describes a gene that determines whether a specialized cell conserves the capacity to become a stem cell again. Unveiling the genetic traits that favour the retention of stem cell properties is crucial for regenerative medicine. Published in Cell Reports, the article is the fruit of collaboration between researchers at IRB Barcelona and CSIC.

One kind of stem cell, those referred to as 'facultative', form parttogether with other cellsof tissues and organs. There is apparently nothing that differentiates these cells from the others. However, they have a very special characteristic, namely they retain the capacity to become stem cells again. This phenomenon is something that happens in the liver, an organ that hosts cells that stimulate tissue growth, thus allowing the regeneration of the organ in the case of a transplant. Knowledge of the underlying mechanism that allows these cells to retain this capacity is a key issue in regenerative medicine.

Headed by Jordi Casanova, research professor at the Instituto de Biologa Molecular de Barcelona (IBMB) of the CSIC and at IRB Barcelona, and by Xavier Franch-Marro, CSIC tenured scientist at the Instituto de Biologa Evolutiva (CSIC-UPF), a study published in the journal Cell Reports reveals a mechanism that could explain this capacity. Working with larval tracheal cells of Drosophila melanogaster, these authors report that the key feature of these cells is that they have not entered the endocycle, a modified cell cycle through which a cell reproduces its genome several times without dividing.

"The function of endocycle in living organisms is not fully understood," comments Xavier Franch-Marro. "One of the theories is that endoreplication contributes to enlarge the cell and confers the production of high amounts of protein". This is the case of almost all larval cells of Drosophila.

The scientists have observed that the cells that enter the endocycle lose the capacity to reactivate as stem cells. "The endocycle is linked to an irreversible change of gene expression in the cell," explains Jordi Casanova, "We have seen that inhibition of endocycle entry confers the cells the capacity to reactivate as stem cells".

Cell entry into the endocycle is associated with the expression of the Fzr gene. The researchers have found that inhibition of this gene prevents this entry, which in turn leads to the conversion of the cell into an adult progenitor that retains the capacity to reactivate as a stem cell. Therefore, this gene acts as a switch that determines whether a cell will enter mitosis (the normal division of a cell) or the endocycle, the latter triggering a totally different genetic program with a distinct outcome regarding the capacity of a cell to reactivate as a stem cell.

Explore further: Autophagy helps fast track stem cell activation

More information: Specification of Differentiated Adult Progenitors via Inhibition of Endocycle Entry in the Drosophila Trachea, Nareg J.-V. Djabrayan, Josefa Cruz, Cristina de Miguel, Xavier Franch-Marro, Jordi Casanova, Cell Reports (2014) DOI: dx.doi.org/10.1016/j.celrep.2014.09.043

In spite of considerable research efforts around the world, we still do not know the determining factors that confer stem cells their main particular features: capacity to self-renew and to divide and proliferate. The scientist ...

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A mechanism that allows a differentiated cell to reactivate as a stem cell revealed

The U.S. Food and Drug Administration today announced it has awarded 15 grants totaling more than $19 million to boost the development of medical device, drug, and biological products for patients with rare diseases, with at least a quarter of the funding going to studies focused solely on pediatrics.

The FDA awards grants for clinical studies on safety and/or effectiveness of products that could either result in, or substantially contribute to, approval of the products.

The FDA is in a unique position to help those who suffer from rare diseases by offering several important incentives to promote the development of products for rare diseases, one of which is this grants program, said Gayatri R. Rao, M.D., director of the FDAs Office of Orphan Product Development. The grants awarded this year support much-needed research in difficult-to-treat diseases that have little, or no, available treatment options.

The program is administered through the FDAs Orphan Products Grants Program. This program was created by the Orphan Drug Act, passed in 1983, to promote the development of products for rare diseases. Since its inception, the program has given more than $330 million to fund more than 530 new clinical studies on developing treatments for rare diseases and has been used to bring more than 50 products to marketing approval.

A panel of independent experts with experience in the disease-related fields reviewed the grant applications and made recommendations to the FDA.

The 2014 grant recipients are:

For the grants program therapies, a disease or condition is considered rare if it affects less than 200,000 persons in the United States. There are about 7,000 rare diseases and conditions, according to the National Institutes of Health. In total, nearly 30 million Americans suffer from at least one rare disease.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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FDA awards grants to stimulate drug, device development for rare diseases

Professor Ryan Lister says he is humbled by the award.

A scientist from the University of WA says he is humbled to be awarded the Prime Minister's prize for life science.

Professor Ryan Lister researches epigenomes - the chemical compounds surrounding DNA - and is one of six people to receive a prize for science from Prime Minister Tony Abbott in Canberra.

Professor Lister has mapped how genes are turned on and off, revealing why a leaf cell is different from a root cell or a stem cell different from a skin cell.

He said he hoped his research could be used to improve the understanding of the human brain, transform stem-cell medicine and advance agriculture.

"We need to be able to understand how the different cell types of our bodies form and how they form in healthy states, so that we can understand why they might be disturbed in various disease states," Professor Lister said.

He said the epigenome played a pivotal role in normal development and disease or stress states in humans, animals and plants.

"What we've been able to do is create the first maps of how the brain epigenome changes during development," he said.

"What this will allow us to do in the future is to look at a range of neurological disorders to see whether these chemical signposts added to the DNA are changed or disturbed or altered within these various disease states.

"We're also researching how the epigenome might affect plant development and the growth and health of crops.

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UWA scientist Ryan Lister wins Prime Minister's prize for life science

Q: You recently wrote about stem cell research, and I understand that stem cell therapy is being used to treat inflammatory bowel disease in cats. Do you have more details?

A: The Winn Feline Foundation has funded the research of Dr. Craig Webb and Dr. Tracy Webb of Colorado State University College of Veterinary Medicine to study the use of stem cells to treat inflammatory bowel disease in cats. Early results are promising.

Stem cell research in cats doesn't stop there. Dr. Glenn Olah, president of the Winn Feline Foundation, notes that Winn also funded stem cell studies to treat feline asthma and kidney disease. Results are hopeful, but it's simply too early to offer definitive answers.

"In some ways, stem cell studies in pets are ahead of (those in) people."

Q: About a month ago, I adopted a beautiful Burmese after she romanced me at the shelter. Once we got home, she wanted nothing to do with me. It's not that she isn't friendly. She loves my son and even sleeps with him. When I get up early to feed her, she stays away until I've left the room. My son suggested that the cat harbors resentment toward me because I took her from her cat friends. What can do to improve the situation?

A: "The good news is that it's very unlikely the cat harbors any resentment," said Winn Feline board member and feline veterinarian Dr. Drew Weigner, of Atlanta. "The bad news for you -- but good news for the cat and your son -- is that they developed a fast friendship.

Here are tips that might help the cat warm up to you:

Sit on the floor in an empty room with her. Close the door, but provide an empty box or two for the cat to hop into. Then, simply watch TV, or read a children's story out loud. Cats sometimes like that soft sing-song voice we tend to use when reading children's stories.

Wait until the cat comes to you. It may take several days, but eventually curiosity will out.

Next, take over feeding the cat, even if she waits for you to leave the room to eat.

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My Pet World: Stem cell treatments show promise for some feline health issues

The bone marrow and stem cell transplant program at the Siteman Cancer Center is one of the largest in the world, completing nearly 500 transplants each year and more than 5,000 since 1982. The program has performed unrelated donor transplants since 1992.

Our physicians use the latest clinical techniques and resources to collect stem cells or peripheral blood for allogeneic transplants, in which transplanted cells come from siblings and unrelated donors. By manipulating stem cell grafts, they also are working to reduce tumor contamination and bolster immunity. Whenever it is appropriate, they recommend that patients participate inclinical trials, research studies that test whether new ways to prevent, diagnose and treat cancer are safe and effective.

At any given time, Siteman offers more than 40 therapeutic clinical trials for patients with leukemia, lymphoma, multiple myeloma and related disorders, including studies that incorporate transplant. Our large patient population allows us to offer single-institution studies and provides us with access to a wide range of tissue samples for future study.

In recent years, Siteman physicians have conducted clinical studies that led to the approval of the drug plerixafor to mobilize, or harvest, stem cells for transplant in patients with non-Hodgkin lymphoma and multiple myeloma. They participated in studies that showed decitabine and high-dose lenalidomide were effective treatments for elderly patients with acute myelogenous leukemia (AML). And they were the first to use a novel suicide gene for gene therapy to control graft-versus-host disease, a serious complication of transplantation.

Dedicated facilities include a 26-bed unit for patients undergoing transplant, which offers eight ICU beds and special HEPA filtration systems to reduce the risk of infection and a second unit for transplant patients and those with blood-related cancers, currently licensed for 38 beds.

Our program has long been an active member of theNational Marrow Donor Program, International Bone Marrow Transplant Registry, North American Bone Marrow Transplant Registry, Blood and Marrow Transplant Clinical Trials Network and Cancer and Leukemia Group B (CALGB) Transplant Consortium.

Excerpt from:
Bone Marrow Transplant and Stem Cell Transplant Program ...

The long-term goal of Dr. Ikeda's lab is to develop efficient and safe gene and cell therapy platforms for individualized medicine. Dr. Ikeda's main research interests include induced pluripotent stem (iPS) cell technology as a novel diabetes therapy; adeno-associated virus (AAV) vector-mediated gene therapy for diabetes and cardiovascular disease; and intrinsic immunity against HIV and retroviral infection.

Towards patient-specific iPS cells for a novel cell therapy for type I diabetes

Dr. Ikeda's research interests include:

Gene and cell therapy for diabetes. Induced pluripotent stem (iPS) cell technology enables derivation of pluripotent stem cells from nonembryonic sources. Successful differentiation of autologous iPS cells into islet-like cells could allow in vitro modeling of patient-specific disease pathogenesis and future clinical cell therapy for diabetes. However, an efficient methodology is not available for the generation of glucose-responsive insulin-producing cells from iPS cells in vitro.

Recently, the lab has examined the efficiency of iPS differentiation into glucose-responsive insulin-producing cells using a modified stepwise protocol with indolactam V and GLP-1 and demonstrated successful generation of islet-like cells, which expressed pancreas-specific markers. Importantly, the iPS-derived islet-like cells secreted C peptide in a glucose-dependent manner. The lab is currently working on reprogramming diabetic patient-derived cells into genomic modification-free iPS cells using nonintegrating vectors, as well as studying the therapeutic effects of iPS-derived insulin-producing islet-like cells in a diabetic mouse model.

Additionally, the lab has developednovel pancreatic gene delivery vectors and is currently studying the therapeutic effects of pancreatic overexpression of factors known to accelerate beta cell regeneration and neogenesis in diabetic mouse models.

Gene therapy for hypertensive heart disease. Altered myocardial structure and function secondary to hypertensive heart disease are leading causes of heart failure and death. A frequent clinical phenotype of cardiac disease is diastolic dysfunction associated with high blood pressure, which over time leads to profound cardiac remodeling, fibrosis and progression to congestive heart failure.

B-type natriuretic peptide (BNP) has blood pressure lowering, anti-fibrotic and anti-hypertrophic properties, making it an attractive therapeutic for attenuating the adverse cardiac remodeling associated with hypertension. However, use of natriuretic peptides for chronic therapy has been limited by their extremely short in vivo half-life. Recently, the lab usedmyocardium-tropic adeno-associated virus serotype 9 (AAV9)-based vectors and demonstrated long-term cardiac BNP expression in spontaneous hypertensive rats. Sustained BNP expression significantly lowered blood pressure for up to nine months and improved the cardiac functions in hypertensive heart disease.

The lab is currently examining the feasibility of this strategy in a large animal model for future clinical applications, as well as further developing a gene therapy strategy for hypertensive heart disease using other therapeutic genes.

Pathogenesis of HIV and retroviruses. Mammalian cells have evolved several strategies to limit viral production. For instance, type 1 interferons stimulate a series of cellular factors that block viral gene expression by degrading viral RNA or inhibiting protein translation.

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Overview Gene and Cell Therapy for Diabetes and ...

21 hours ago

In a study that will provide the foundation for scientists to better replicate natural stem cell development in an artificial environment, UCLA researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research led by Dr. Guoping Fan, professor of human genetics, have established a benchmarking standard to assess how culture conditions used to procure stem cells in the lab compare to those found in the human embryo.

The study was published online ahead of print in the journal Cell Stem Cell.

Pluripotent stem cells (PSCs) are cells that can transform into almost any cell in the human body. Scientists have long cultured PSCs in the laboratory (in vitro) using many different methods and under a variety of conditions. Though it has been known that culture techniques can affect what kind of cells PSCs eventually become, no "gold standard" has yet been established to help scientists determine how the artificial environment can better replicate that found in a natural state (in vivo).

Dr. Kevin Huang, postdoctoral fellow in the lab of Dr. Fan and a lead author of the study, analyzed data from multiple existing research studies conducted over the past year. These previously published studies used different culture methods newly developed in vitro in the hopes of coaxing human stem cells into a type of pluripotency that is in a primitive or ground-zero state.

Utilizing recently-published gene expression profiles of human preimplantation embryos as the benchmark to analyze the data, Dr. Huang and colleagues found that culture conditions do affect how genes are expressed in PSCs, and that the newer generation culture methods appear to better resemble those found in the natural environment of developing embryos. This work lays the foundation on the adoption of standardized protocol amongst the scientific community.

"By making an objective assessment of these different laboratory techniques, we found that some may have more of an edge over others in better replicating a natural state," said Dr. Huang. "When you have culture conditions that more consistently match a non-artificial environment, you have the potential for a much better reflection of what is going on in actual human development."

With these findings, Dr. Fan's lab hopes to encourage further investigation into other cell characteristics and molecular markers that determine the effectiveness of culture conditions on the proliferation and self-renewal of PSCs.

"We hope this work will help the research community to reach a consensus to quality-control human pluripotent stem cells," said Dr. Fan.

Explore further: Technique to make human embryonic stem cells more closely resemble true epiblast cells

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Team proposes benchmark to better replicate natural stem cell development in the laboratory environment

San Diego, California (PRWEB) October 28, 2014

The top stem cell clinic in San Diego, Telehealth, is now offering regenerative medicine procedures for the knee to help restore cartilage and avoid the need for joint replacement. The procedures are outpatient and performed by Board Certified doctors at Telehealth. Call (888) 828-4575 for more information and scheduling.

Hundreds of thousands of knee replacements are performed every year in the US, with most being extremely successful. However, it is a major surgery and there is a chance of complications such as infection or blood clot. Therefore, it is advisable to consider a stem cell procedure for the arthritic knee in an effort to delay or avoid the procedure.

Telehealth provides the procedures with several options, including platelet rich plasma therapy, bone marrow or fat derived stem cells, along with amniotic derived procedures. All of the procedures are outpatient and low risk.

In most cases, the procedures are covered in whole or partly by insurance. Telehealth will perform an insurance verification prior to one's procedure. The Board Certified doctors at the stem cell clinic in San Diego treat patients from a broad area in Southern California. There are several locations including La Jolla, Orange and Upland CA.

In addition to stem cell procedures for knee arthritis, TeleHealth also provides regenerative medicine options for tendon and ligament injuries, sports injuries along with hip, shoulder and ankle arthritis.

For those interested in avoiding knee replacement with a procedure that can potentially preserve or repair arthritic cartilage, call Telehealth at (888) 828-4575 and visit http://stemcelltherapyincalifornia.com/ for more information.

Link:
San Diego Stem Cell Clinic, Telehealth, Now Offering Knee Procedures for Cartilage Restoration

In 2010, Darek Fidyka was paralyzed from the chest down as a result of a knife attack that left an 8 mm gap in his spinal column. Now surgeons in Poland, working in collaboration with scientists in London, have given Fidyka the ability to walk again thanks to a new procedure using transplanted cells from his olfactory bulbs.

The spinal injury that left Darek Fidyka paralyzed did not see the spinal cord entirely severed, but rather an 8 mm chunk removed from the left side. Researchers have for years worked to develop treatments to help those with spinal injuries, but for Fidyka no amount of therapy was helping him recover feeling below his chest. Now, two years after the groundbreaking treatment, Fidyka has regained some feeling in his legs, feet, bowels, bladder, and can now walk with the assistance of a frame.

The procedure saw the medical team remove one of Fidykas olfactory bulbs then grow olfactory ensheathing cells (OECs) in culture and graft the cells onto his damaged spinal column where they helped to re-link vital nerve fibers. According to the UCL, the OECs act as pathway cells that repair and renew nerve fibers when damaged. The team chose OECs as they are the only part of the nervous system with the ability to regenerate in adults.

A few weeks after the initial OEC removal and culture harvesting, the team applied 100 micro-injections of the olfactory cells above and below the injured area. Then four thin strips of nerve tissue from Fidykas ankle were applied across the damaged area. After about three months they noticed muscle mass increasing on his left thigh, and after six months Fidyka was able to stand and take his first steps with the assistance of parallel bars, leg braces and a physiotherapist. Today he still undergoes five hours of physiotherapy, five days a week.

"It is immensely gratifying to see that years of research have now led to the development of a safe technique for transplanting cells into the spinal cord." said Professor Geoff Raisman, Chair of Neural Regeneration at the UCL Institute of Neurology. "I believe we stand on the threshold of a historic advance and that the continuation of our work will be of major benefit to mankind. I believe we have now opened the door to a treatment of spinal cord injury that will get patients out of wheel chairs. Our goal now is to develop this first procedure to a point where it can be rolled out as a worldwide general approach."

The BBC Panorama program To Walk Again shows the procedure and footage of Fidyka walking with a frame. When asked what it was like to walk again, Fidyka said, "when you cant feel almost half your body, you are helpless, but when it starts coming back its as if you were born again."

The treatment marks a world first in cell transplantation and paralysis reversal. The project was jointly funded by the Nicholls Spinal Injury Foundation and the UK Stem Cell Foundation. Professor Raisman, who first discovered OECs in 1985, went on to show how the treatment could be applied on rats with spinal injuries in 1997.

Details of the research can be found in the journal Cell Transplantation.

Sources: UCL Institute of Neurology, BBC Panorama

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Cell transplant enables paralyzed man to walk again

10 hours ago

The New York Stem Cell Foundation (NYSCF) Research Institute, through the launch of its repository in 2015, will provide for the first time the largest-ever number of stem cell lines available to the scientific research community. Initially, over 600 induced pluripotent stem (iPS) cell lines and 1,000 cultured fibroblasts from over 1,000 unique human subjects will be made available, with an increasing number available in the first year. To collect these samples, NYSCF set up a rigorous human subjects system that protects patients and allows for the safe and anonymous collection of samples from people interested in participating in research.

A pilot of over 200 of NYSCF's iPS cell lines is already searchable on an online database. The pilot includes panels of iPS cell lines generated from donors affected by specific diseases such as type 1 diabetes, Parkinson's disease, and multiple sclerosis, as well as a diversity panel of presumed healthy donors from a wide range of genetic backgrounds representing the United States Census. These panels, curated to provide ideal initial cohorts for studying each area, include subjects ranging in age of disease onset, and are gender matched. Other panels that will be available in 2015 include Alzheimer's disease, schizophrenia, Juvenile Batten disease, and Charcot-Marie-Tooth disease.

"NYSCF's mission is to develop new treatments for patients. Building the necessary infrastructure and making resources available to scientists around the world to further everyone's research are critical steps in accomplishing this goal," said Susan L. Solomon, CEO of The New York Stem Cell Foundation.

NYSCF has developed the technology needed to create a large collection of stem cell lines representing the world's population. This platform, known as the NYSCF Global Stem Cell ArrayTM, is an automated robotic system for stem cell production and is capable of generating 200 iPS cell lines a month from patients with various diseases and conditions and from all genetic backgrounds. The NYSCF Global Stem Cell ArrayTM is also used for stem cell differentiation and drug screening.

Currently available in the online database that was developed in collaboration with eagle-i Network, of the Harvard Catalyst, is a pilot set of approximately 200 iPS cell lines and related information about the patients. This open source, open access resource discovery platform makes the cell lines and related information available to the public on a user-friendly, web-based, searchable system. This is one example of NYSCF's efforts to reduce duplicative research and enable even broader collaborative research efforts via data sharing and analysis. NYSCF continues to play a key role in connecting the dots between patients, scientists, funders, and outside researchers that all need access to biological samples.

"The NYSCF repository will be a critical complement to other existing efforts which are limited in their ability to distribute on a global scale. I believe that this NYSCF effort wholly supported by philanthropy will help accelerate the use of iPS cell based technology," said Dr. Mahendra Rao, NYSCF Vice President of Regenerative Medicine.

To develop these resources, NYSCF has partnered with over 50 disease foundations, academic institutions, pharmaceutical companies, and government entities, including the Parkinson's Progression Markers Initiative (PPMI), PersonalGenomes.org, the Beyond Batten Disease Foundation, among several others. NYSCF also participates in and drives a number of large-scale multi stakeholder initiatives including government and international efforts. One such example is the Cure Alzheimer's Fund Stem Cell Consortium, a group consisting of six institutions, including NYSCF, directly investigating, for the first time, brain cells in petri dishes from individual patients who have the common sporadic form of Alzheimer's disease.

"We are entering this next important phase of using stem cells to understand disease and discover new drugs. Having collaborated with NYSCF extensively over the last five years on the automation of stem cell production and differentiation, it's really an exciting moment to see these new technologies that NYSCF has developed now being made available to the entire academic and commercial research communities," said Dr. Kevin Eggan, Professor of Stem Cell and Regenerative Biology at Harvard University and Principal Investigator of the Harvard Stem Cell Institute.

NYSCF's unique technological resources have resulted in partnerships with companies to develop both stem cell lines and also collaborative research programs. Over the past year, NYSCF has established collaborations with four pharmaceutical companies to accelerate the translation of basic scientific discoveries into the clinic. Federal and state governments are also working with NYSCF to further stem cell research in the pursuit of cures. In 2013, NYSCF partnered with the National Institutes of Health (NIH) Undiagnosed Disease Program (UDP) to generate stem cell lines from 100 patients in the UDP and also collaborate with UDP researchers to better understand and potentially treat select rare diseases.

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NYSCF Research Institute announces largest-ever stem cell repository

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Newswise Scientists have described a way to convert human skin cells directly into a specific type of brain cell affected by Huntingtons disease, an ultimately fatal neurodegenerative disorder. Unlike other techniques that turn one cell type into another, this new process does not pass through a stem cell phase, avoiding the production of multiple cell types, the studys authors report.

The researchers, at Washington University School of Medicine in St. Louis, demonstrated that these converted cells survived at least six months after injection into the brains of mice and behaved similarly to native cells in the brain.

Not only did these transplanted cells survive in the mouse brain, they showed functional properties similar to those of native cells, said senior author Andrew S. Yoo, PhD, assistant professor of developmental biology. These cells are known to extend projections into certain brain regions. And we found the human transplanted cells also connected to these distant targets in the mouse brain. Thats a landmark point about this paper.

The work appears Oct. 22 in the journal Neuron.

The investigators produced a specific type of brain cell called medium spiny neurons, which are important for controlling movement. They are the primary cells affected in Huntingtons disease, an inherited genetic disorder that causes involuntary muscle movements and cognitive decline usually beginning in middle-adulthood. Patients with the condition live about 20 years following the onset of symptoms, which steadily worsen over time.

The research involved adult human skin cells, rather than more commonly studied mouse cells or even human cells at an earlier stage of development. In regard to potential future therapies, the ability to convert adult human cells presents the possibility of using a patients own skin cells, which are easily accessible and wont be rejected by the immune system.

To reprogram these cells, Yoo and his colleagues put the skin cells in an environment that closely mimics the environment of brain cells. They knew from past work that exposure to two small molecules of RNA, a close chemical cousin of DNA, could turn skin cells into a mix of different types of neurons.

In a skin cell, the DNA instructions for how to be a brain cell, or any other type of cell, is neatly packed away, unused. In past research published in Nature, Yoo and his colleagues showed that exposure to two microRNAs called miR-9 and miR-124 altered the machinery that governs packaging of DNA. Though the investigators still are unraveling the details of this complex process, these microRNAs appear to be opening up the tightly packaged sections of DNA important for brain cells, allowing expression of genes governing development and function of neurons.

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Human Skin Cells Reprogrammed Directly Into Brain Cells

I have to admit that my first response to these reports out of Britain that stem cells had been successfully used to repair a complete spinal cord transection was skepticism incredulity even. Theyre reporting that a man with a completely severed spinal cord at level T10-T11 is able to walk again! The Guardian gushes! The Daily Mail gets in the act (always a bad sign)! When I read that the patient had an 8mm gap in his spinal cord that had been filling up with scar tissue for the last two years, I was even more doubtful: under the best of conditions, it was unlikely that youd get substantial connectivity across that distance.

So I read the paper. Im less skeptical now, for a couple of reasons. They actually did this experiment on 3 people, and all showed degrees of improvement, although the newspapers are all focusing on just the one who had the greatest change. The gradual changes are all documented thoroughly and believably. And, sad to say, the improvements in the mans motor and sensory ability are more limited and more realistic than most of the accounts would have you think.

The story is actually in accord with what weve seen in stem cell repair of spinal cord injury in rats and mice.

Overall, they found that stem cell treatment results in an average improvement of about 25% over the post-injury performance in both sensory and motor outcomes, though the results can vary widely between animals. For sensory outcomes the degree of improvement tended to increase with the number of cells introduced scientists are often reassured by this sort of dose response, as it suggests a real underlying biologically plausible effect. So the good news is that stem cell therapy does indeed seem to confer a statistically significant improvement over the residual ability of the animals both to move and feel things beyond the spinal injury site.

Significant but far from complete improvement is exactly what wed expect, and that improvement is a very, very good thing. It is an accomplishment to translate animal studies into getting measurable clinical improvements in people.

The basic procedure is straightforward. There is a population of neural cells in humans that do actively and continuously regenerate: the cells of the olfactory bulb. So what they did is remove one of the patients own olfactory bulbs, dissociate it into a soup of isolated cells, and inject them into locations above and below the injury. They also bridged the gap with strips of nerve tissue harvested from the patients leg. The idea is that the proliferating cells and the nerves would provide a nerve growth-friendly environment and build substrate bridges that would stimulate the damaged cells and provide a path for regrowth.

Big bonus: this was an autologous transplant (from the patients own tissues), so there was no worry about immune system rejection. There were legitimate worries about inflammation, doing further damage to the spinal cord, and provoking greater degeneration, and part of the purpose of this work was to assess the safety of the procedure. There were no complications.

Also, Im sure you were worried about this, but the lost olfactory cells also regenerated and the patients completely recovered their sense of smell.

Now heres the clinical assessment. Three patients were operated on; T1 is the one who has made all the news with the most remarkable improvement. There were also three control patients who showed no improvement over the same period.

Neurological function improved in all three transplant recipients (T1, T2, T3) during the first year postsurgery. This included a decrease of muscle spasticity (T1, T2) as well as improvement of sensory (T1, T2, T3) and motor function (T1, T2, T3) below the level of spinal cord injury.

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Stem cell treatment of spinal cord injuries [Pharyngula]

A man paralysed from the waist down after his spinal cord was sliced in half in a stabbing attack is able to walk again thanks to cells transplanted from his NOSE.

Darek Fidyka, 38, is believed to be the first person in the world to recover from complete severing of the spinal nerves.

The Bulgarian - who suffered his injury in 2010 - can now walk with a frame and has been able to resume an independent life, even to the extent of driving a car.

Sensation has returned to his lower limbs.

Surgeons used nerve-supporting cells from Darek's nose to provide pathways along which the broken tissue was able to grow.

Despite success in the laboratory, it is the first time the procedure has been shown to work in a human patient.

Professor Geoffrey Raisman, whose team at University College London's Institute of Neurology discovered the technique, said: "We believe that this procedure is the breakthrough which, as it is further developed, will result in a historic change in the currently hopeless outlook for people disabled by spinal cord injury."

The research, funded by the Nicholls Spinal Injury Foundation (NSIF) and the UK Stem Cell Foundation, is featured in a special Panorama programme on BBC One tonight.

A Polish team led by one of the world's top spinal repair experts, Dr Pawel Tabakow, from Wroclaw Medical University, performed the surgery.

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Paralysed man able to walk again thanks to cells transplanted from his NOSE

PUBLIC RELEASE DATE:

20-Oct-2014

Contact: David McKeon 212-365-7440 New York Stem Cell Foundation @nyscf

Leaders in translational stem cell research from around the world will present the latest advances in stem cell science that are leading to better treatments and cures to disease and injury at The New York Stem Cell Foundation's Ninth Annual Translational Stem Cell Research Conference.

The opening day of the conference includes a panel discussion on large scale, big data stem cell and genetic initiatives moderated by Susan L. Solomon, JD, CEO and Co-founder of The New York Stem Cell Foundation (NYSCF), with panelists George Church, PhD, Harvard Medical School; John Greally, PhD, Albert Einstein College of Medicine; Scott Noggle, PhD, The NYSCF Research Institute; and Eric Schadt, PhD, the Icahn School of Medicine at Mount Sinai.

Later that day, a discussion on neurodegeneration includes Kevin Eggan, PhD, Harvard University and the NYSCF Research Institute, who will discuss his research identifying an existing drug candidate that may be of use treating ALS and is entering clinical trials in the coming year. The following session on cell reprogramming and cancer includes Michael Milone, MD, PhD, University of Pennsylvania, who will discuss recent research results from his lab and his colleagues including the results of a clinical trial for leukemia featured in The New York Times last week. The first day closes with a conversation on personalized medicine featuring Dieter Egli, PhD, NYSCF Robertson Investigator at the NYSCF Research Institute and Columbia University; Rudolf Jaenisch, MD, The Whitehead Institute; and Sir Ian Wilmut, FRS, FRSE, University of Edinburgh.

On October 23, the day will begin with remarks by Kenneth Adams and Kyle Kimball, President of the Empire State Development Corporation and President of the New York City Economic Development Corporation, respectively. The session on translating innovation from the laboratory to the clinic features Stephen Chang, PhD, of the NYSCF Research Institute and Richard Pearse, PhD, of the Harvard Catalyst and eagle-i Network who will discuss their collaboration on the first publicly available induced pluripotent stem cell database. The day will close with a presentation on induced neuronal cells and cell transdifferentiation from the 2014 NYSCF Robertson Stem Cell Prize recipient, Marius Wernig, MD, PhD, of Stanford University School of Medicine.

Sir Ian Wilmut will give the keynote address on October 22nd and Dr. Rudolf Jaenisch will give the keynote address on the last day of the conference.

The full conference agenda can be found at http://www.nyscf.org/conference

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Stem cell and clinical research advances to be presented at NYSCF's Ninth Annual Conference

Antonio Regalado for MIT Technology Review 2014-10-15 19:15:44 UTC

When stem cells were first culled from human embryos sixteen years ago, scientists imagined they would soon be treating diabetes, heart disease, stroke, and many other diseases with cells manufactured in the lab.

It's all taken longer than they thought. But now, a Massachusetts biotech firm has reported results from the largest, and longest, human test of a treatment based on embryonic stem cells, saying it appears safe and may have partly restored vision to patients going blind from degenerative diseases.

Results of three-year study were described Tuesday in the Lancet by Advanced Cell Technology and collaborating eye specialists at the Jules Stein Eye Institute in Los Angeles who transplanted lab-grown cells into the eyes of nine people with macular degeneration and nine with Stargardt's macular dystrophy.

The idea behind Advanced Cell's treatment is to replace retinal pigment epithelium cells, known as RPE cells, a type of caretaker tissue without which a person's photoreceptors also die, with supplies grown in laboratory. It uses embryonic stem cells as a starting point, coaxing them to generate millions of specialized retina cells. In the study, each patient received a transplant of between 50,000 and 150,000 of those cells into one eye.

The main objective of the study was to prove the cells were safe. Beyond seeing no worrisome side effects, the researchers also noted some improvements in the patients. According to the researchers half of them improved enough to read two to three extra lines on an eye exam chart, results Robert Lanza, chief scientific officer of Advanced Cell, called remarkable.

"We have people saying things no one would make up, like 'Oh I can see the pattern on my furniture, or now I drive to the airport," he says. "Clearly there is something going on here."

Lanza stressed the need for a larger study, which he said the company hoped to launch later this year in Stargardt's patients. But if the vision results seen so far continue, Lanza says "this would be a therapy."

Some eye specialists said it's too soon to say whether the vision improvements were real. The patients weren't examined by independent specialists, they said, and eyesight in patients with low vision is notoriously difficult to measure. That leaves plenty of room for placebo effects or unconscious bias on the part of doctors.

"When someone gets a treatment, they try really hard to read the eye chart," says Stephen Tsang, a doctor at Columbia University who sees patients losing their vision to both diseases. It's common for patients to show quick improvements, he says, although typically not as large as what Advanced Cell is reporting.

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Stem Cell Eye Treatment May Restore Vision

PUBLIC RELEASE DATE:

14-Oct-2014

Contact: David McKeon DMcKeon@nyscf.org 212-365-7440 New York Stem Cell Foundation @nyscf

NEW YORK, NY (October 14, 2014) The New York Stem Cell Foundation (NYSCF) announced today that Marius Wernig, PhD, Associate Professor in the Institute for Stem Cell Biology and Regenerative Medicine and the Department of Pathology at Stanford University School of Medicine, is the 2014 recipient of the NYSCF Robertson Stem Cell Prize, which has been awarded since 2011 for extraordinary achievements in translational stem cell research by a young scientist.

Dr. Wernig and his team discovered that human skin cells can be converted directly into functional neurons, termed induced neuronal (iN) cells, in a period of four to five weeks with the addition of just four proteins.

"Dr. Wernig's groundbreaking research has the potential to accelerate all research on devastating neurodegenerative diseases," said Susan L. Solomon, CEO and Co-founder of NYSCF. "His work can impact and accelerate research on multiple sclerosis, Alzheimer's disease, and autism among many other conditions."

At Stanford, Dr. Wernig focuses on using induced pluripotent stem (iPS) cells and iN cells for disease modeling and as potential cellular therapy. This new technique transformed the field of cellular reprogramming by eliminating the need to first create iPS cells, making it easier to generate patient or disease-specific neurons. These cell types hold tremendous therapeutic and translational relevance for patients around the world. Potential applications range from replacing damaged brain tissue to repairing the myelinating nerves lost in multiple sclerosis to identifying novel drugs and treatments for a range of neurological diseases.

In addition to his recent scientific achievements, Dr. Wernig was part of the inaugural class of NYSCF Robertson Stem Cell Investigators in 2010, and is the first NYSCF Robertson Investigator to receive the NYSCF Robertson Stem Cell Prize.

"I am delighted that Dr. Wernig is being recognized with this year's NYSCF Robertson Prize for his important research that has opened entirely new avenues for studying brain diseases. The NYSCF Robertson Prize was created to acknowledge the most important work being down by young stem cell scientists and I am thrilled to see a NYSCF Robertson Investigator go on to receive NYSCF Robertson Prize," said Julian Robertson, whose foundation underwrites the $200,000 prize. The terms of the prize require that the $200,000 stipend be used, at the recipients' discretion, to further support their research.

The NYSCF Robertson Stem Cell Prize will be presented to Dr. Wernig at a ceremony in New York City by Susan L. Solomon on October 14th.

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Marius Wernig receives New York Stem Cell Foundation's Robertson Stem Cell Prize

PUBLIC RELEASE DATE:

13-Oct-2014

Contact: Robert Miranda cogcomm@aol.com Cell Transplantation Center of Excellence for Aging and Brain Repair

Putnam Valley, NY. (Oct. 13, 2014) Two studies recently published in Cell Transplantation reveal that cell transplantation may be an effective treatment for spinal cord injury (SCI), a major cause of disability and paralysis with no current restorative therapies.

Using laboratory rats modeled with SCI, researchers in Spain found in laboratory tests on cells harvested from rats - specifically ependymal progenitor cells (epSPCs), multipotent stem cells found in adult tissues surrounding the ependymal canal of the spinal cord - responded to a variety of compounds through the activation of purinergic receptors P2X4, P2X7, P2Y1 and P2Y4. In addition, the epSPCs responded to adenosine triphosphate (ATP) through this activation. ATP, a chemical produced by a wide variety of enzymes that works to transport energy within cells, is known to accumulate at the sites of spinal cord injury and cooperate with growth factors that induce remodeling and repair.

"The aim of our study was to analyze the expression profile of receptors in ependymal-derived neurospheres and to determine which receptors were functional by analysis of intercellular Ca2+ concentration," said study co-author Dr. Rosa Gomez-Villafuertes of the Department of Biochemistry at the Veterinary School at the University of Complutense in Madrid, Spain. "We demonstrated for the first time that epSPCs express functional ionotropic P2X4 and P2X7 and metabotropic P2Y1 and P2Y4 receptors that are able to respond to ATP, ADP and other nucleotide compounds."

When they compared the epSPCs from healthy rats to epSPCs from rats modeled with SCI, they found that a downregulation of P2Y1 and an upregulation of P2Y4 had occurred in the epSPCs in the SCI group.

"This finding opens an important avenue for potential therapeutic alternatives in SCI treatments based on purinergic receptor modulation," the researchers concluded.

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The study will be published in a future issue of Cell Transplantation and is currently freely available on-line as an unedited early e-pub at: http://www.ingentaconnect.com/content/cog/ct/pre-prints/content-CT-1257_Gomez_Villafuertes.

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Spinal cord injury victims may benefit from stem cell transplantation studies

Researchers have developed a technique to jump-start the body's systems for creating blood vessels, opening the door for potential new treatments for diseases whose impacts include amputation and blindness.

The international team, led by scientists at the Indiana University School of Medicine, is targeting new therapies for illnesses such as peripheral artery disease, a painful leg condition caused by poor blood circulation. The disease can lead to skin problems, gangrene and sometimes amputation.

While the body has cells that specialize in repairing blood vessels and creating new ones, called endothelial colony-forming cells, these cells can lose their ability to proliferate into new blood vessels as patients age or develop diseases like peripheral arterial disease, said Mervin C. Yoder Jr., M.D., Richard and Pauline Klingler Professor of Pediatrics at IU and leader of the research team.

Peripheral artery disease patients can be given medication to improve blood flow, but if the blood vessels to carry that improved flow are reduced in number or function, the benefits are minimal. If "younger," more "enthusiastic" endothelial colony forming cells could be injected into the affected tissues, they might jump-start the process of creating new blood vessels. Gathering those cells would not be easy however -- they are relatively difficult to find in adults, especially in those with peripheral arterial disease. However, they are present in large numbers in umbilical cord blood.

Reporting their work in the journal Nature Biotechnology, the researchers said they had developed a potential therapy through the use of patient-specific induced pluripotent stem cells, which are normal adult cells that have been "coaxed" via laboratory techniques into reverting into the more primitive stem cells that can produce most types of bodily tissue. So, in one of the significant discoveries reported in the Nature Biotechnology paper, the research team developed a novel methodology to mature the induced pluripotent stem cells into cells with the characteristics of the endothelial colony-forming cells that are found in umbilical cord blood. Those laboratory-created endothelial colony-forming cells were injected into mice, where they were able to proliferate into human blood vessels and restore blood flow to damaged tissues in mouse retinas and limbs.

Overcoming another hurdle that has been faced by scientists in the field, the research team found that the cord-blood-like endothelial colony-forming cells grown in laboratory tissue culture expanded dramatically, creating 100 million new cells for each original cell in a little less than three months.

"This is one of the first studies using induced pluripotent stem cells that has been able to produce new cells in clinically relevant numbers -- enough to enable a clinical trial," Dr. Yoder said. The next steps, he said, include reaching an agreement with a facility approved to produce cells for use in human testing. In addition to peripheral artery disease, the researchers are evaluating the potential uses of the derived cells to treat diseases of the eye and lungs that involve blood flow problems.

Story Source:

The above story is based on materials provided by Indiana University. Note: Materials may be edited for content and length.

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New cells meant to form blood vessels developed, treat peripheral artery disease

Suzanne Kreiter/Boston Globe/Getty

A research team led by Douglas Melton (left) has made insulin-secreting cells using human stem cells.

Each year, surgeon Jose Oberholzer frees a few people with type1 diabetes from daily insulin injections by giving them a transplant of the insulin-secreting -cells that the disease attacks. But it is a frustrating process. Harvested from a cadavers pancreas, the -cells are in short supply and vary in quality. And the patients must take drugs to suppress their immune response to the foreign cells, which can in turn cause kidney failure.

On 9October, stem-cell researcher Douglas Melton of Harvard University in Cambridge, Massachusetts, and his colleagues reported an advance that has the potential to overcome Oberholzers frustrations and allow many more people with type1 diabetes to receive transplants. Melton and his team have achieved a long-term goal of stem-cell science: they have created mature -cells using human stem cells that can be grown from a potentially unlimited supply, and that behave like the real thing (F.W.Pagliuca etal. Cell 159, 428439; 2014). The next challenge is to work out how to shield these -cells from the bodys immune response.

Researchers had previously created immature -cells from stem cells and transplanted them into diabetic mice. But they take months to mature into insulin-secreting cells, and it is unclear whether they would do so in humans.

The -cells reported by Meltons team were grown from adult cells that had been reprogrammed to resemble stem cells. In response to glucose, the -cells quickly secreted insulin, which the body uses to regulate blood sugar. When implanted in diabetic mice, the cells relieved symptoms within two weeks. The -cells even formed clusters that are similar to those found in a pancreatic structure called the islet of Langerhans. If you took these cells and showed them to somebody without telling them what they are, I guarantee you an expert would say that is a perfect human islet cell, says Oberholzer, who is working with Meltons team to test the cells in non-human primates.

A remaining hurdle is shielding the cells from immune attack. This is necessary if the treatment is to become more widely available, because immunosuppressant drugs can be justified only in the most severe cases of diabetes. And although mature -cells could be derived from a patients own skin cells, type1 diabetes is an autoimmune disease, so transplanted cells would still be vulnerable to attack.

One solution might be to encapsulate the cells in a credit-card-sized, biocompatible sheath made by ViaCyte of San Diego, California. The company will implant its first device loaded with immature -cells in a patient on 21October. Studies in animals have been promising, but some researchers worry that the cells inside the device are packed too densely and might become starved of oxygen and nutrients.

Another option is to coat cells in a protective hydrogel, which results in thousands of separate balls of cells. But a potential drawback is that it would be much harder to remove such cells if there was a safety concern, says Albert Hwa, director of discovery science at JDRF, a diabetes-research foundation in New York.

Neither technique avoids the bodys tendency to enclose foreign bodies inside scar tissue, which could cut the transplanted cells off from nutrients. Bioengineer Daniel Anderson of the Massachusetts Institute of Technology in Cambridge and his team are screening chemical compounds for a hydrogel that does not trigger this. Some, used with Meltons cells, have shown promise in unpublished studies of diabetic primates, he says.

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Stem-cell success poses immunity challenge for diabetes