Archive for the ‘Cell Medicine’ Category

Australian scientists studying zebrafish have stumbled upon what they say is one of the most significant discoveries in stem cell research.

In research published on Thursday in the journal Nature, the Monash University scientists revealed that they uncovered how one of the most important stem cells in blood and bone marrow, the haematopoietic stem cell (HSC), is formed.

Professor Peter Currie, from Monash University's Australian Regenerative Medicine Institute, said the discovery brought researchers closer to growing HSCs in a lab.

"HSCs are the basis of bone marrow transplantations as a therapy, so when a leukaemia patient receives bone marrow, it's really these HSCs that do the heavy lifting," Professor Currie said.

"So when clinicians do bone marrow transplants, they need to find a matching donor recipients and we know that's a hit-or-miss procedure.

"So for many years people have been trying to make HSCs in the dish, and they've had very little success in doing this."

Professor Currie, who led the study, said the discovery brought scientists much closer to achieving that aim.

"It's the discovery of a completely new cell type that basically is required to give instructions to the HSC to make it become what it needs to become," he said.

"It means we now understand how HSC form in the body better, we can use that information to try to grow these cells in the dish and we hope that will lead to better treatment for people with leukaemia and blood disorders."

Professor Currie said he specialises in muscle stem cell biology and accidentally came across the discovery while studying muscle stem cells in zebrafish.

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Stem cell discovery: Australian scientists make significant find while studying zebrafish

PUBLIC RELEASE DATE:

13-Aug-2014

Contact: Lucy Handford media@monash.edu Monash University

A cure for a range of blood disorders and immune diseases is in sight, according to scientists who have unravelled the mystery of stem cell generation.

The Australian study, led by researchers at the Australian Regenerative Medicine Institute (ARMI) at Monash University and the Garvan Institute of Medical Research, is published today in Nature. It identifies for the first time mechanisms in the body that trigger hematopoietic stem cell (HSC) production.

Found in the bone marrow and in umbilical cord blood, HSCs are critically important because they can replenish the body's supply of blood cells. Leukemia patients have been successfully treated using HSC transplants, but medical experts believe blood stem cells have the potential to be used more widely.

Lead researcher Professor Peter Currie, from ARMI explained that understanding how HSCs self-renew to replenish blood cells is a "Holy Grail" of stem cell biology.

"HSCs are one of the best therapeutic tools at our disposal because they can make any blood cell in the body. Potentially we could use these cells in many more ways than current transplantation strategies to treat serious blood disorders and diseases, but only if we can figure out how they are generated in the first place. Our study brings this possibility a step closer," he said.

A key stumbling block to using HSCs more widely has been an inability to produce them in the laboratory setting. The reason for this, suggested from previous research, is that a molecular 'switch' may also be necessary for HSC formation, though the mechanism responsible has remained a mystery, until now.

In this latest study, ARMI researchers observed cells in the developing zebra fish - a tropical freshwater fish known for its regenerative abilities and optically clear embryos - to gather new information on the signalling process responsible for HSC generation.

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Cell discovery brings blood disorder cure closer

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Newswise Researchers at the University of California, San Diego School of Medicine have launched a clinical trial to investigate the safety of neural stem cell transplantation in patients with chronic spinal cord injuries. This Phase I clinical trial is recruiting eight patients for the 5-year study.

The goal of this study is to evaluate the safety of transplanting neural stem cells into the spine for what one day could be a treatment for spinal cord injuries, said Joseph Ciacci, MD, principal investigator and neurosurgeon at UC San Diego Health System. The studys immediate goal, however, is to determine whether injecting these neural stem cells into the spine of patients with spinal cord injury is safe.

Related goals of the clinical trial include evaluating the stem cell grafts survival and the effectiveness of immunosuppression drugs to prevent rejection. The researchers will also look for possible therapeutic benefits such as changes in motor and sensory function, bowel and bladder function, and pain levels.

Patients who are accepted for the study will have spinal cord injury to the T7-T12 level of the spines vertebrae and will have incurred their injury between one and two years ago.

All participants will receive the stem cell injection. The scientists will use a line of human stem cells approved by the U.S. FDA for human trials in patients with chronic traumatic spinal injuries. These cells were previously tested for safety in patients with amyotrophic lateral sclerosis (ALS).

Since stem cell transplantation for spinal cord injury is just beginning clinical tests, unforeseen risks, complications or unpredictable outcomes are possible. Careful clinical testing is essential to ensure that this type of therapy is developed responsibly with appropriate management of the risks that all medical therapies may present.

Pre-clinical studies of these cells by Ciacci and Martin Marsala, MD, at the UC San Diego School of Medicine, showed that these grafted neural stem cells improved motor function in spinal cord injured rats with minimal side effects indicating that human clinical trials are now warranted.

This clinical trial at UC San Diego Health System is funded by Neuralstem, Inc. and was launched and supported by the UC San Diego Sanford Stem Cell Clinical Center. The Center was recently created to advance leading-edge stem cell medicine and science, protect and counsel patients, and accelerate innovative stem cell research into patient diagnostics and therapy.

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Clinical Trial Evaluates Safety of Stem Cell Transplantation in Spine

Miami (PRWEB) August 10, 2014

Regenestem, a division of the Global Stem Cells Group, Inc., has announced the launch of a new stem cell treatment center in Cozumel, Mexico, offering the most advanced protocols and techniques in cellular medicine to patients from around the world.

A team of stem cell medical professionals led by Rafael Moguel, M.D., an advocate and pioneer in the use of stem cell therapies to treat a range of medical conditions, will provide cutting edge therapies and follow-up treatment under the Regenestem brand.

In June, Global Stem Cells Group opened the Regenestem Asia Clinic in Manila, Philippines, adding a new state-of-the-art regenerative medicine facility to the company's growing global presence that includes clinics in Miami, New York, Los Angeles, and Dubai. Regenestem Asia facility marks the first Regenestem brand clinic in the Philippines.

Regenestem provides stem cell treatments for a variety of diseases and conditions, including arthritis, autism, chronic obstructive pulmonary disease (COPD), diabetes, and multiple sclerosis at various facilities worldwide. Regenestem Mexico will have an international staff experienced in administering the leading cellular therapies available.

Regenestem Mexico is certified for the medical tourism market, and staff physicians are board-certified or board-eligible. Regenestem clinics provide services in more than 10 specialties, attracting patients from the United States and around the world.

The Global Stem Cells Group and Regenestem are committed to the highest of standards in service and technology, expert and compassionate care, and a philosophy of exceeding the expectations of their international patients.

For more information, visit the Regenestem website, email info(at)regenstem(dot)com, or call 305-224-1858.

About Regenestem:

Regenestem, a division of the Global Stem Cells Group, Inc., is an international medical practice association committed to researching and producing comprehensive stem cell treatments for patients worldwide. Having assembled a highly qualified staff of medical specialistsprofessionals trained in the latest cutting-edge techniques in cellular medicineRegenestem continues to be a leader in delivering the latest protocols in the adult stem cell arena.

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Global Stem Cells Group and Regenestem Announce Launch of Stem Cell Treatment Center in Cozumel, Mexico

A new first-of-its kind pilot study has revealed that stem cell treatment can significantly improve recovery from stroke in humans.

The therapy uses a type of cell called CD34+ cells, a set of stem cells in the bone marrow that give rise to blood cells and blood vessel lining cells. Rather than developing into brain cells themselves, the cells are thought to release chemicals that trigger the growth of new brain tissue and new blood vessels in the area damaged by stroke.

The patients were treated within seven days of a severe stroke, in contrast to several other stem cell trials, most of which have treated patients after six months or later. The Imperial researchers believe early treatment might improve the chances of a better recovery.

Dr Soma Banerjee, Consultant in Stroke Medicine at Imperial College Healthcare NHS Trust, said that the treatment appeared to be safe and that it's feasible to treat patients early when they might be more likely to benefit.

However, it's too early to draw definitive conclusions about the effectiveness of the therapy and more tests to work out the best dose and timescale for treatment before starting larger trials, she further added.

The study is published in the journal Stem Cells Translational Medicine.

(Posted on 09-08-2014)

Original post:
Stem cell treatment holds hope for better stroke recovery

UC San Francisco researchers have identified cells' unique features within the developing human brain, using the latest technologies for analyzing gene activity in individual cells, and have demonstrated that large-scale cell surveys can be done much more efficiently and cheaply than was previously thought possible.

"We have identified novel molecular features in diverse cell types using a new strategy of analyzing hundreds of cells individually," said Arnold Kriegstein, MD, PhD, director of the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UCSF. "We expect to use this approach to help us better understand how the complexity of the human cortex arises from cells that are spun off through cell division from stem cells in the germinal region of the brain."

The research team used technology focused on a "microfluidic" device in which individual cells are captured and flow into nano-scale chambers, where they efficiently and accurately undergo the chemical reactions needed for DNA sequencing. The research showed that the number of reading steps needed to identify and spell out unique sequences and to successfully identify cell types is 100 times fewer than had previously been assumed. The technology, developed by Fluidigm Corporation, can be used to individually process 96 cells simultaneously.

"The routine capture of single cells and accurate sampling of their molecular features now is possible," said Alex Pollen, PhD, who along with fellow Kriegstein-lab postdoctoral fellow Tomasz Nowakowski, PhD, conducted the key experiments, in which they analyzed the activation of genes in 301 cells from across the developing human brain. Their results were published online August 3 in Nature Biotechnology.

Kriegstein said the identification of hundreds of novel biomarkers for diverse cell types will improve scientists' understanding of the emergence of specialized neuronal subtypes. Ultimately, the combination of this new method of focusing on gene activity in single cells with other single-cell techniques involving microscopic imaging is likely to reveal the origins of developmental disorders of the brain, he added.

The process could shed light on several brain disorders, including lissencephaly, in which the folds in the brain's cortex fail to develop, as well as maladies diagnosed later in development, such as autism and schizophrenia, Kriegstein said.

According to the Nature Biotechnology study co-authors, this strategy of analyzing molecules in single cells is likely to find favor not only among researchers who explore how specialized cells arise at specific times and locations within the developing organism, but also among those who monitor cell characteristics in stem cells engineered for tissue replacement, and those who probe the diversity of cells within tumors to identify those responsible for survival and spread of cancerous cells.

No matter how pure, in any unprocessed biological sample there are a variety of cells representing various tissue types. Researchers have been sequencing the combined genetic material within these samples. To study which genes are active and which are dormant, they use the brute repetition of sequencing steps to capture an adequate number of messenger RNA sequences, which are transcribed from switched-on genes. However, it is difficult to conclude from mixed tissue samples which genes are expressed by particular cell types.

Pollen and Nowakowski showed that fewer steps -- and less time and money -- are needed to distinguish different cell types through single-cell analysis than had previously been thought.

"We are studying an ecosystem of different, but related, cell types in the brain," Pollen said. "We are breaking that community down into the different populations of cells with the goal of understanding their functional parts and components so we can accurately predict how they will develop."

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Single-cell analysis holds promise for stem cell and cancer research

PUBLIC RELEASE DATE:

6-Aug-2014

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

Putnam Valley, NY. (Aug. 6, 2014) Recent studies have shown that transplanting induced pluripotent stem cell-derived neural stem cells (iPS-NSCs) can promote functional recovery after spinal cord injury in rodents and non-human primates. However, a serious drawback to the transplantation of iPS-NSCs is the potential for tumor growth, or tumorogenesis, post-transplantation.

In an effort to better understand this risk and find ways to prevent it, a team of Japanese researchers has completed a study in which they transplanted a human glioblastoma cell line into the intact spinal columns of laboratory mice that were either immunodeficient or immunocompetent and treated with or without immunosuppresant drugs. Bioluminescent imaging was used to track the transplanted cells as they were manipulated by immunorejection.

The researchers found that the withdrawal of immunosuppressant drugs eliminated tumor growth and, in effect, created a 'safety lock' against tumor formation as an adverse outcome of cell transplantation. They also confirmed that withdrawal of immunosuppression led to rejection of tumors formed by transplantation of induced pluripotent stem cell derived neural stem/progenitor cells (iPS-NP/SCs).

Although the central nervous system has shown difficulty in regenerating after damage, transplanting neural stem/progenitor cells (NS/PCs) has shown promise. Yet the problem of tumorogenesis, and increases in teratomas and gliomas after transplantation has been a serious problem. However, this study provides a provisional link to immune therapy that accompanies cell transplantation and the possibility that inducing immunorejection may work to reduce the likelihood of tumorogenesis occurring.

"Our findings suggest that it is possible to induce immunorejection of any type of foreign-grafted tumor cells by immunomodulation," said study co-author Dr. Masaya Nakamura of the Keio University School of Medicine. "However, the tumorogenic mechanisms of induced pluripotent neural stem/progenitor cells (iPS-NS/PCs) are still to be elucidated, and there may be differences between iPS-NS/PCs derived tumors and glioblastoma arising from genetic mutations, abnormal epigenetic modifications and altered cell metabolisms."

The researchers concluded that their model might be a reliable tool to target human spinal cord tumors in preclinical studies and also useful for studying the therapeutic effect of anticancer drugs against malignant tumors.

"This study provides evidence that the use of, and subsequent removal of, immunosuppression can be used to modulate cell survival and potentially remove tumor formation by transplanted glioma cells and provides preliminary data that the same is true for iPS-NS/PCs." said Dr. Paul Sanberg, distinguished professor at the Center of Excellence for Aging and Brain Repair, University of South Florida. "Further study is required to determine if this technique could be used under all circumstances where transplantation of cells can result in tumor formation and its reliability in other organisms and paradigms."

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Researchers seek 'safety lock' against tumor growth after stem cell transplantation

A method of growing human cells from tissue removed from a patient's gastrointestinal (GI) tract eventually may help scientists develop tailor-made therapies for inflammatory bowel disease and other GI conditions.

Reporting online recently in the journal Gut, researchers at Washington University School of Medicine in St. Louis said they have made cell lines from individual patients in as little as two weeks. They have created more than 65 such cell lines using tissue from 47 patients who had routine endoscopic screening procedures, such as colonoscopies. A cell line is a population of cells in culture with the same genetic makeup.

The scientists said the cell lines can help them understand the underlying problems in the GI tracts of individual patients and be used to test new treatments.

"While it has been technically possible to isolate intestinal epithelial stem cells from patients, it has been challenging to use the material in ways that would benefit them on an individual basis," said co-senior investigator Thaddeus S. Stappenbeck, MD, PhD, a professor of pathology and immunology. "This study advances the field in that we have developed new methods that allow for the rapid expansion of intestinal epithelial stem cells in culture. That breaks a bottleneck and allows us to develop new ways to test drug and environmental interactions in specific patients."

To grow the human cells, the researchers adapted a system used to grow intestinal epithelial stem cells in mice. In the GI tract, epithelial cells line the inner surface of the esophagus, stomach and intestines.

"An additional important feature of this system is that we can isolate stem cell lines from intestinal biopsies," said first author Kelli L. VanDussen, PhD, a postdoctoral fellow in Stappenbeck's laboratory. "These biopsies are very small tissue fragments that are routinely collected by a gastroenterologist during endoscopy procedures. We have refined this technique, so we have nearly 100 percent success in creating cell lines from individual patient biopsies."

The researchers developed an experimental system that created high levels of critical factors to isolate and expand intestinal epithelial stem cells, including a signaling protein called Wnt and a related protein called R-spondin, which enhances the Wnt signal. They also exposed the cells to a protein called Noggin, which prevented the cells from differentiating into other cell types that live in the GI tract.

After growing the intestinal cell lines, the investigators collaborated with Phillip I. Tarr, MD, the Melvin E. Carnahan Professor of Pediatrics and director of the Division of Pediatric Gastroenterology and Nutrition, to conduct experiments and see how the cells interacted with bacterial pathogens like E. coli.

This showed that pathogenic strains of E. coli attached to intestinal epithelial cells. That attachment is thought to be the critical step in stimulating disease. The investigators said the experimental system they created should lead to new methods to uncover therapies for treating bacterial infections of the intestine.

"In the past, the only really robust method for studying GI epithelial cells was to use cancer cell lines," said co-senior investigator Matthew A. Ciorba, MD, a gastroenterologist and assistant professor of medicine. "However, cancer cells behave differently than the noncancerous GI epithelium, which is affected in patients with conditions such as inflammatory bowel disease. This technique now allows us to study cells identical to the ones that live in a patient's GI tract. Plus, we can grow the cell lines quickly enough that it should be possible to develop a personalized approach to understanding a patient's disease and to tailor treatment based on a patient's underlying problem."

Continued here:
Growing human GI cells may lead to personalized treatments

By Partnership for Public Service August 5

Dr. Griffin Rodgers spends most of his waking hours leading the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), but he also manages to carve out time to work on a life-long passion discovering a cure for sickle cell disease.

Long before becoming the director of NIDDK, Rodgers was credited with discovering the first effective therapy for sickle cell disease, an inherited blood disorder that affects more than 90,000 Americans, most of them African-Americans. The disease, which affects millions of people throughout the world, can damage bones, joints and internal organs, cause acute and chronic pain, and often result in premature death.

Prior to his discovery of a drug treatment in the 1990s, the only options for sickle cell patients were blood transfusions for pain and supportive care.

This initial breakthrough has been followed by the recent announcement that Rodgers and a team of National Institutes of Health (NIH) researchers have developed a modified blood stem-cell transplant regimen that is highly effective in reversing sickle cell disease in adults. The findings, based on a clinical trial of 30 patients, represent a potentially transformative treatment.

Dr. Neal Young, chief of NIHs Hematology Branch of the National Heart, Lung and Blood Institute, said Rodgers has been the driving force behind the advanced medical treatments for people with sickle cell disease. His work, said Young, is a very big deal because it will save the lives and alleviate the suffering of thousands of people.

Dr. Thomas Starzl, a physician and researcher who performed the worlds first liver transplant, wholeheartedly concurred.

Griffin Rodgers work on sickle cell disease has been revolutionary, said Starzl. I can only give him rave reviewsfive stars.

Rodgers grew up in New Orleans where he had three high school friends who became debilitated with sickle cell disease. Two of those friends died in their teenage years and the third passed away a few years after high school.

These deaths left a tremendous impression on Rodgers, who pursued a medical career that led him to NIH in 1984 where he began his work on sickle cell disease. Over the years as he made his mark in the laboratory and the clinical setting, Rodgers also progressed through the managerial ranks, heading NIDDKs Molecular and Clinical Hematology Branch starting in 1998, becoming deputy director of NIDDK in 2001 and director of the institute in 2007.

More:
NIH scientist transforming treatment of sickle cell disease

Time Posted: August 5, 2014 9:43 am

Yanick Mulumba

Kabungo Yanick Mulumba, a resident of Maamba, Zambia, a graduate of St. Canisius High School, and now a senior at Harvard University in Cambridge, Massachusetts, USA, is one of forty undergraduate students accepted into the 2014 Harvard Stem Cell Institute (HSCI) Internship Program, which provides participants with a challenging summer research experience in a cutting-edge stem cell science laboratory.

Mulumba is spending ten weeks, from June 9 to August 15, in the Harvard University Department of Stem Cell and Regenerative Biology laboratory of HSCI Principal Faculty member Chad Cowan, PhD, known for his research on genetic disease modeling. Mulumbas project this summer is to engineer transplantable white blood cells that dont attack the bodys own cells when used for adoptive immunotherapya treatment that uses biological substances to boost a patients immune system.

The internship has enhanced my critical thinking through troubleshooting and planning of experiments, Mulumba said. Ive also been exposed to leaders in academia and industry who have helped me learn how to combine my interests in medicine, research, and healthcare management.

Over the course of the program, interns participate in a stem cell seminar series, a career pathways presentation, and a weekly stem cell companion course. They present their summer research findings, both orally and in poster format, at an end-of-program symposium.

This program represents an exciting opportunity for undergraduates to gain hands-on experience in stem cell research while working in an HSCI laboratory under the supervision of an experienced researcher, said HSCI Internship Program co-director M. William Lensch, PhD.

The Harvard Stem Cell Institute gratefully acknowledges the generous support of the following sponsors for the 2014 HSCI Internship Program: Biogen Idec, Boehringer Ingelheim Pharmaceuticals Inc., EPSRC Centre for Innovative Manufacturing in Regenerative Medicine, Loughborough University (UK), Novartis Institutes for BioMedical Research, and Vertex Pharmaceuticals.

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Maamba resident awarded Harvard Stem cell institute internship

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Dr. Joseph Purita is a pioneer within the worldwide orthopaedic surgery community. He has lectured on five continents and has been instrumental in helping some countries design their policies concerning the use of regenerative medicine. Dr. Purita graduated from Georgetown University Medical School and completed his residency at University of Miami-Jackson Memorial Hospital. Like all Stem.MD physicians, Dr. Purita prides himself on offering the latest surgical and non-surgical techniques to our clients, which range from celebrities to weekend athletes to the elderly. Read more about Stem MD.

[youtube]http://www.youtube.com/watch?v=8H5oxvt6Gt4[/youtube]

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Stem.MD | National Regenerative Medical Practice

By Amy Norton HealthDay Reporter

WEDNESDAY, July 30, 2014 (HealthDay News) -- Babies born with so-called "bubble boy" disease can often be cured with a stem cell transplant, regardless of the donor -- but early treatment is critical, a new study finds.

Severe combined immunodeficiency (SCID), as the condition is medically known, actually refers to a group of rare genetic disorders that all but eliminate the immune system. That leaves children at high risk of severe infections.

The term "bubble boy" became popular after a Texas boy with SCID lived in a plastic bubble to ward off infections. The boy, David Vetter, died in 1984 at the age of 12, after an unsuccessful bone marrow transplant -- an attempt to give him a functioning immune system.

Today, children with SCID have a high chance of survival if they receive an early stem cell transplant, researchers report in the July 31 issue of the New England Journal of Medicine.

In the best-case scenario, a child would get stem cells -- the blood-forming cells within bone marrow -- from a sibling who is a perfect match for certain immune-system genes.

But that's not always an option, partly because kids with SCID are often their parents' first child, said Dr. John Cunningham, director of hematopoietic stem cell transplantation at the University of Chicago Comer Children's Hospital. He was not involved in the study.

In those cases, doctors typically turn to a parent -- who is usually a "half" match, but whose stem cells can be purified to improve the odds of success. Sometimes, stem cells from an unrelated, genetically matched donor can be used.

The good news: Regardless of the donor, children with SCID can frequently be cured, according to the new findings. But early detection and treatment is vital.

"These findings show that if you do these transplants early -- before [the age of] 3.5 months, in a child without infection -- the results are really quite comparable to what you have with a matched sibling," said lead researcher Dr. Richard O'Reilly, chief of the pediatric bone marrow transplant service at Memorial Sloan-Kettering Cancer Center in New York City.

Continued here:
Early Stem Cell Transplant Vital in 'Bubble Boy' Disease

PUBLIC RELEASE DATE:

31-Jul-2014

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research

Advances in stem cell research will provide enormous opportunities for both biological and future clinical applications. Basically, stem cells could replicate any other cells in the body, offering immense hope of curing Alzheimer's disease, repairing damaged spinal cords, treating kidney, liver and lung diseases and making damaged hearts whole. The potential for profit is staggering. Prof. Jinhui Chen from Indiana University in USA considered that this field of research still faces myriad biological, ethical, legal, political, and financial challenges. The eventual resolution of these conflicts will determine the success of the research and potentially the face of medicine in the future. The relevant study has been published in the Neural Regeneration Research (Vol. 9, No. 7, 2014).

###

Article: " A brief review of recent advances in stem cell biology " by Jinhui Chen1, Libing Zhou2, Su-yue Pan3 (1 Stark Neuroscience Research Institute and Department of Neurological Surgery, Indiana University School of Medicine, Indianapolis, IN, USA; 2 Guangdong-Hongkong-Macau Institute of CNS Regeneration (GHMICR), Jinan University, Guangzhou, Guangdong Province, China; 3 Department of Neurology, Nanfang Hospital, Southern Medical University, Guangzhou, Guangdong Province, China)

Chen JH, Zhou LB, Pan SY. A brief review of recent advances in stem cell biology. Neural Regen Res.2014;9(7):684-687.

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research http://www.nrronline.org/

AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert! system.

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Recent advances in stem cell biology

Boston, MA (PRWEB) July 31, 2014

Bostons Adult Stem Cell Technology Center, LLC (ASCTC) finds itself flush with innovative adult stem cell biotechnologies. Currently the company holds seven recently issued patents and has three additional patent applications currently under examination by the U.S. Patent and Trademarks Office.

The patented inventions address two of the most vexing problems in adult stem cell biology research and regenerative medicine. Adult stem cells are difficult to identify; and they have been difficult to multiply to sufficient numbers to support regenerative medicine applications.

ASCTC has addressed the identity problem by developing patented biomarkers that are found exclusively on adult stem cells. The biomarkers are based on ASCTCs expertise in defining properties of adult stem cells that are not shared by any other normal cell types in the body. The patented biomarkers also identify some types of cancer stem cells. Therefore, they have applications in both stem cell medicine and cancer medicine.

ASCTCa success in developing procedures for producing adult stem cells in large numbers is due to the companys expertise in adult stem cell growth control. ASCTCs technology uses natural compounds found in the body to instruct adult stem cells to multiply in a controlled manner as during normal body growth.

The companys patented method for controlling adult stem cells to multiply without losing their stem cell properties has applications for many different types of adult stem cells. ASCTCs approved patents demonstrate the application of the method for production of human liver stem cells, hair follicle stem cells, and human pancreatic stem cells; but the technology has general application to adult stem cells found in many other types of organs and tissues.

In addition to the main focus on adult stem cell technologies, ASCTCs most recently issued patent applies its cell multiplication methods to produce induced pluripotent stem cells (iPSCs) without transferring exogenous genes. This gene-free single agent method should offer significant value to the many mushrooming companies that supply iPSCs and iPSC production reagents.

As a small start-up, ASCTC is employing a social media marketing strategy. In the past week, the company has launched patent licensing ads on LinkedIn, Vocus, and Facebook, as well increased its advertising references within its recently established Twitter presence.

It would be a shame for these technologies to lie dormant, just because our hands are full with other projects at the moment. James Sherley, director of ASCTC, relates that the companys two main business efforts require only a fraction of its available intellectual property. ASCTC is currently focused on bringing laboratory-scale production of human liver stem cells to manufacturing scales and developing a computer simulation assay for preclinical detection of drug candidates with intolerable toxicity due to adverse effects on adult stem cells.

Sherley adds, We already have a few companies that have expressed interest in licensing. But we could do a lot better at reaching others whose development efforts would benefit from ASCTCs unique technologies. Love to hear from ViaCyte!

Read more here:
The Adult Stem Cell Technology Center, LLC Launches A Marketing Campaign To License Adult Stem Cell Biotechnologies

"Bubble boy" David Vetter lived in a protective environment designed by NASA engineers. He died of complications after receiving a bone marrow transplant in 1984, at the age of 12. Baylor College of Medicine Photo Archives

Babies born with so-called "bubble boy" disease can often be cured with a stem cell transplant, regardless of the donor -- but early treatment is critical, a new study finds.

Severe combined immunodeficiency (SCID), as the condition is medically known, actually refers to a group of rare genetic disorders that all but eliminate the immune system. That leaves children at high risk of severe infections.

The term "bubble boy" became popular after a Texas boy with SCID lived in a plastic bubble to ward off infections. The boy, David Vetter, died in 1984 at the age of 12, after an unsuccessful bone marrow transplant -- an attempt to give him a functioning immune system.

15 Photos

Immune disorder forced David Vetter to live in bubble - but breakthroughs from his story now enable similar kids to live free

In the best-case scenario, a child would get stem cells -- the blood-forming cells within bone marrow -- from a sibling who is a perfect match for certain immune-system genes.

But that's not always an option, partly because kids with SCID are often their parents' first child, said Dr. John Cunningham, director of hematopoietic stem cell transplantation at the University of Chicago Comer Children's Hospital. He was not involved in the study.

In those cases, doctors typically turn to a parent -- who is usually a "half" match, but whose stem cells can be purified to improve the odds of success. Sometimes, stem cells from an unrelated, genetically matched donor can be used.

The good news: Regardless of the donor, children with SCID can frequently be cured, according to the new findings. But early detection and treatment is vital.

See original here:
Early stem cell transplant may cure "bubble boy" disease

A new stem-cell discovery might one day lead to a more streamlined process for obtaining stem cells, which in turn could be used in the development of replacement tissue for failing body parts, according to UC San Francisco scientists who reported the findings in the current edition of Cell.

The work builds on a strategy that involves reprogramming adult cells back to an embryonic state in which they again have the potential to become any type of cell.

The efficiency of this process may soon increase thanks to the scientists identification of biochemical pathways that can inhibit the necessary reprogramming of gene activity in adult human cells. Removing these barriers increased the efficiency of stem-cell production, the researchers found.

Our new work has important implications for both regenerative medicine and cancer research, said Miguel Ramalho-Santos, Ph.D., associate professor of obstetrics, gynecology and reproductive sciences and a member of the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UCSF, who led the research, funded in part by a prestigious NIH Directors New Innovator Award.

The earlier discovery that it was possible to take specialized adult cells and reverse the developmental clock to strip the mature cells of their distinctive identities and characteristics and to make them immortal, reprogrammable cells that theoretically can be used to replace any tissue type led to a share of the Nobel Prize in Physiology or Medicine being awarded to UCSF, Gladstone Institutes and Kyoto University researcher Shinya Yamanaka, M.D., in 2012.

These induced pluripotent stem (iPS) cells are regarded as an alternative experimental approach to ongoing efforts to develop tissue from stem cells obtained from early-stage human embryos. However, despite the promise of iPS cells and the excitement surrounding iPS research, the percentage of adult cells successfully converted to iPS cells is typically low, and the resultant cells often retain traces of their earlier lives as specialized cells.

Researchers generate stem cells by forcing the activation within adult cells of pluripotency-inducing genes starting with the so-called Yamanaka factors a process that turns back the clock on cellular maturation.

Yet, as Ramalho-Santos notes, From the time of the discovery of iPS cells, it was appreciated that the specialized cells from which they are derived are not a blank slate. They express their own genes that may resist or counter reprogramming.

But the nature of what exactly was getting in the way of reprogramming remained poorly understood. Now, by genetically removing multiple barriers to reprogramming, we have found that the efficiency of generation of iPS cells can be greatly increased, he said. The discovery will contribute to accelerating the safe and efficient use of iPS cells and other reprogrammed cells, according to Ramalho-Santos.

Miguel Ramalho-Santos

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Stem cell discovery may make tissue regeneration more efficient

Released: 29-Jul-2014 8:00 AM EDT Source Newsroom: Mayo Clinic Contact Information

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Newswise JACKSONVILLE, Fla. Imagine a future in which a new lung is grown for a patient in need, using the patients own cellular material, or a day when an injection of replacement cells will enable a patient to self-heal damage in the brain, nerves or other tissues.

MULTIMEDIA ALERT: For audio and video of Dr. Keller and Jorge Bacardi talking about the gift and regenerative medicine, visit the Mayo Clinic News Network.

Regenerative medicine is no longer science fiction, and a substantial gift from Jorge and Leslie Bacardi of the Bahamas will significantly accelerate the research of Mayo Clinics Center for Regenerative Medicine on the Florida campus.

Jorge Bacardi, whose family has manufactured rum and other spirits for 150 years, suffered since childhood with primary ciliary dyskinesia, a debilitating lung disease that nearly ended his life. A double lung transplant at Mayos Florida campus in 2008 enabled him to take his first full breath of air at age 64.

Regenerative medicine is an extraordinary step in the evolution of mankind, says Jorge Bacardi. It is for Leslie and I a great honor to be able to join Mayo Clinic in the development of such an advancement in the medical field."

Regenerative medicine is addressing the root causes of disease and disability by developing ways to rejuvenate the body using its natural self-healing processes; replace damaged cells with healthy ones derived from the patient (avoiding immune system rejection); and regenerate function by applying specific cells or cell products.

Mayos regenerative medicine researchers are targeting conditions throughout the body, including heart disease, stroke, Alzheimers disease and traumatic injuries that affect combat veterans. Some studies are in the earliest stages. Others are in clinical trials with patients.

Researchers now can differentiate stem cells into skin, brain, lung and many other types of cells. For example, a patient's own skin cells may be collected, reprogrammed in a laboratory to give them certain characteristics, and then delivered back to the patient to treat diseases at various places within the body.

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Bacardis Make Gift to Significantly Advance Mayo Clinic's Regenerative Medicine Research

Tampa Bay, FL (PRWEB) July 28, 2014

Nearly 53 million Americans today are suffering with arthritis, with the majority of them diagnosed with osteoarthritis. (1) Osteoarthritis is a degeneration of joint cartilage and its underlying bone, causing significant pain and stiffness. While osteoarthritis has no cure, stem cell therapy has been demonstrated to induce profound healing in many forms of arthritis, according to the Stem Cell Institute. (2) Dr. Cynthia Elliott of Skinspirations, a center for cosmetic enhancement devoted to non-surgical aesthetics and now also specializing in administering regenerative medicine by stem cell, has made use of these services in a recent case study, which resulted in improved health in one of their clients.

Stem cells are unique from other cells for the following reasons:

(a)They can renew themselves through cell division; and (b)Under certain conditions, they can become tissue or organ-specific cells.

Stem cells are revered for their ability to make replacement tissues, as it relates to regenerative therapy. (3) Medical scientists and researchers are discovering the seemingly endless possibilities of what stem cells can treat, including brain damage, bone repair, kidney disease, etc. (4) This treatment is starting to boom in the medical world as a viable procedure, but Skinspirations has already had these practices in place, establishing them as progressive practitioners in the field.

Skinspirations is specifically studying the Stromal Vascular Fraction (SVF)another term for stem cell treatmentand how it affects knees with severe arthritis. According to Dr. Elliott, Stromal Vascular Fraction can help to repair, replace and restore any damaged cells within the bodyDr. Elliott performed the stem cell procedure on her uncle after first treating other patients during her training, and he experienced the following results:

Case in Point:

Joe Elliott, a 63-year-old male, had severe arthritis in one knee. Doctors advised him to get a knee replacement, but Joe was hoping to avoid surgery for as long as possible. After talking to Dr. Elliott about the treatment, he drove to Skinspirations from Missouri to go forward with the stem cell procedure.

Dr. Elliott performed the treatment with the following steps:

(1)Numbed his abdomen with anesthesia; (2)Removed about 100 cc of fat; (3)Processed the fat to isolate the SVF; (4)Numbed the arthritic knee; and (5)Injected the pellet of SVF into the joint of his arthritic knee.

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Skinspirations Study Supports Medical Findings: Stem Cell Treatment Triggers Tissue Regeneration

The California Institute for Regenerative Medicine has continued in damage-control mode since the state agencys former president, Alan Trounson, joined the board of directors at StemCells Inc. this month, just seven days after leaving the agency.

Newark-based StemCells has been awarded nearly $20 million in CIRM funding, as part of a long relationship that, in the wake of Trounson's departure, has raised concern about potential conflict of interest.

The agency's new president, C. Randal Mills, said he was taking a strong stand on personal ethics, signing an agreement not to accept a job with any company funded by CIRM for at least one year after leaving his position at the state agency.

"We take even the appearance of conflicts of interest very seriously," Mills said in a statement this month.

But a scientist whose grant proposal was turned down even though it received a higher rating than the StemCells proposal called the relationship between the state agency and the company interesting.

In my opinion, Mr. Trounson and the CIRM staff were clearly antagonistic to us and strongly supportive of StemCells, Lon S. Schneider, a scientist at USCs Keck School of Medicine, told the California Stem Cell Report ,a blog that follows news related to the stem cell agency.

And Times columnist Michael Hiltzik pointed out that the agency has hired its own law firm to conduct the investigation, rather than a completely independent party.

The unanswered question burning a hole through CIRM's credibility is whether StemCells Inc. got its money because its research was promising, or because it knew the right people, Hiltzik wrote.

The stem cell agency has also voted to cut $5 million from a $70-million effort to create a series of statewide stem cell clinics, according to the California Stem Cell Report. And even though the board has 29 members, only eight could vote because of conflicts of interest among the others, according to the report.

Following a thorough review it is my opinion that the $70-million price tag is not clearly justified in terms of the benefits it will deliver to the people of California, Mills wrote in a memo to the agency's board.

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California stem cell agency head takes stand on 'personal ethics'

Stem cell agency President C. Randal Mills (left) and Chairman of the Board Jonathan Thomas.

Responding to his predecessor's ethically controversial departure, the president and chief executive of California's stem cell agency said Thursday he is taking legal steps to minimize conflicts of interests with those who have business before the agency.

C. Randal Mills said he will not take a job with any company funded by the California Institute for Regenerative Medicine for one year after he departs the agency. In addition, he also will not accept gifts or travel payments from any company, institution or person who gets agency funding.

Mills' action, announced at the agency's meeting in Millbrae, will be enforced with a legal agreement he will sign. His action comes less than a month after he replaced Alan Trounson as the agency chief. One week after his departure, CIRM-funded StemCells Inc. announced it had appointed Trounson to its board. StemCells Inc. had received an award of nearly $20 million from the agency to develop a therapy for Alzheimers disease.

While Trounson's appointment wasn't illegal, critics said it was unseemly for him to join a company that had received agency funding so soon after he left CIRM. An ethical controversy could harm the agency's chances of getting more funding from California voters, who gave the agency $3 billion with the passage of Proposition 71 in 2004.

Mills said the new rules apply only to himself, because of his central role at CIRM.

"This specifically addresses an issue where an individual in an organization has a disproportionate amount of power, and I want to make sure it's known that power will not be abused," Mills said.

Mills made the right decision, said Jeanne Loring, a CIRM-funded stem cell researcher at The Scripps Research Institute.

"There's a difference between what is legal and what is ethical," said Loring, who attended the meeting. "And he's going to be pushing the needle a lot more toward the ethical side without worrying whether he can get away with stuff."

John Simpson of Santa Monica-based Consumer Watchdog, who has often criticized CIRM for conflicts of interest, also praised the decision.

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Stem cell agency tightens ethics rules

Published 18 July 2014

ViaCyte a privately held regenerative medicine company developing a cell replacement therapy for the treatment of diabetes, announced that it has filed an Investigational New Drug application (IND) with the United States Food and Drug Administration (FDA) seeking to initiate a Phase 1/2 clinical trial in patients with type 1 diabetes.

The trial would evaluate the safety and efficacy of ViaCyte's VC-01 product candidate, a stem cell-derived, encapsulated cell replacement therapy. In a related development, ViaCyte submitted a Medical Device Master File (called MAF) to the FDA in support of the Encaptra drug delivery system, the device component of the VC-01 product candidate.

"The filing of this IND represents the culmination of many years of research and development by a dedicated team focused on developing a cell replacement therapy for patients with type 1 diabetes and advancing our VC-01 product candidate to human clinical trials," said Paul Laikind, Ph.D., President and Chief Executive Officer of ViaCyte.

"The ViaCyte team has been assisted and supported by the California Institute for Regenerative Medicine (CIRM) a leading organization focused on advancing the field of stem cell-based technologies, and JDRF, the leading advocacy organization for patients with type 1 diabetes," added Dr. Laikind.

ViaCyte's VC-01 product candidate consists of pancreatic progenitor cells, called PEC-01 cells, which are derived from a proprietary human embryonic stem cell line. These cells are then encapsulated by use of ViaCyte's Encaptra device.

When implanted under the skin, the PEC-01 cells are designed to mature and further differentiate into insulin-producing beta and other endocrine cells that regulate blood glucose in a manner similar or identical to the normal islets that comprise the endocrine pancreas.

Based on a pre-IND meeting with the FDA and subsequent consultations, ViaCyte is proposing to initiate clinical evaluation of the VC-01 product candidate directly in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function.

In addition to evaluating the safety of the product candidate in these patients, the study is designed to demonstrate the effectiveness of the VC-01 product candidate in replacing lost endocrine function that is central to the disease.

In the proposed clinical trial, insulin production from the VC-01 implant would be assessed by measuring C-peptide, a biomarker for insulin produced by beta cells that is expected to provide a sensitive measure of efficacy in these patients.

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ViaCyte files investigational new drug application and device master file with FDA for novel cell replacement therapy ...

There's no good time for a public agency to be embroiled in a conflict-of-interest scandal, but this is an especially delicate time for California's stem cell agency.

The California Institute for Regenerative Medicine, as the program is known formally, is on track to finish doling out its $3 billion in funding from the state's voters as soon as 2017. Its original sponsor, Northern California real estate developer Robert Klein II, has been quoted talking about another $5-billion infusion, perhaps via the 2016 ballot.

Any such effort will refocus attention on the program board's inherent conflicts of interest, which were baked in by the terms of Proposition 71, Klein's 2004 ballot initiative that created CIRM and funded it through a bond issue. The prestigious Institute of Medicine in a 2012 report found these conflicts to lead to questions about "the integrity and independence of some of CIRM's decisions."

And now here comes another case. This one involves CIRM former President Alan Trounson, an Australian biologist who left the agency on June 30 and joined the board of one of its highest-profile financial partners a mere seven days later. Trounson's new employer, Stem Cells Inc., is the recipient of a nearly $20-million loan for Alzheimer's research.

CIRM says Trounson's quick move to Stem Cells Inc., where he'll receive a stipend of at least $90,000 a year, is legally "permissible." But officials there acknowledge they were blindsided; the agency learned about Trounson's new position from the company's press release.

Afterward, CIRM rushed out a statement acknowledging that Trounson's appointment to the board of a CIRM loan recipient "creates a serious risk of a conflict of interest." The agency says it will place the relationship between CIRM and the company under "a full review." Administrators reminded Trounson, board members and agency staff that state law bars him from communicating with them on any administrative matter involving Stem Cells Inc. The company declined to comment.

The relationship already reeked of cronyism. As we reported in 2012, the Newark, Calif.-based firm's co-founder, Irving Weissman, director of Stanford University's Institute for Stem Cell Biology and Regenerative Medicine, had been one of the most prominent and outspoken supporters of Proposition 71.

He's also a leading recipient of CIRM funding, listed as the principal investigator on four Stanford grants totaling nearly $35 million. CIRM contributed $43.6 million toward the construction of his institute's $200-million research building at the Stanford campus. Weissman and his wife, Ann Tsukamoto, owned nearly 380,000 shares of the firm as of last April, according to a corporate disclosure. Tsukamoto is one of the company's top executives; Weissman is a board member.

Trounson's move comes as CIRM must begin looking to the future, but any discussions about extending the agency's life span will have to address the flaws created by Proposition 71. Among them is the program's very structure, and even its scientific goals.

Klein's ballot proposition exempts CIRM from virtually any oversight or accountability. Each of the 29 governing board members has to be associated with a California public or private research institution or company, or an advocacy group for patients of one disease or another. The qualifications for board chairman are so specific they initially yielded a single credible candidate: Bob Klein.

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Conflicts of interest pervasive on California stem cell board

Professor John Rasko on SBS Insight
Royal Prince Alfred Hospital #39;s Director of Cell and Molecular Therapies, Professor John Rasko, was invited as a guest on SBS Insight #39;s special on stem cell medicine.

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Professor John Rasko on SBS Insight - Video

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Newswise Learning the role of immune system cells in healthy digestive tracts and how they interact with neighboring nerve cells may lead to new treatments for irritable bowel syndrome (IBS). Researchers from Penn State College of Medicine, in collaboration with other scientists, have reported the role of macrophages in regulating the contractions of the colon to push digested material through the digestive tract.

The muscular lining of the intestine contains a distinct kind of macrophage, an immune system cell that helps fight infections. The role of these cells in normal colon function is not known, although they have been linked to inflammation after abdominal surgery.

Very little is known about the function of muscularis macrophages, mainly because these cells are difficult to isolate from intestinal tissue, said Milena Bogunovic, assistant professor of microbiology and immunology.

Digested material is moved through the intestines by the contraction and relaxation of intestinal muscles. The pattern and frequency of these contractions are controlled by the signals from the intestinal nervous system. In patients with diseases like IBS, the signals are overactive and stimulation is exaggerated.

The researchers developed a method to deplete muscularis macrophages in the intestines of mice to determine their function. They report their findings in Cell.

After macrophage depletion, we observed that the normal intestinal movements are irregular, probably because the muscular contractions were poorly coordinated, suggesting that intestinal movements are regulated by macrophages, Bogunovic said.

After confirming the role of the macrophages in the function of the digestive tract, the researchers looked for how the regulation happens. They compared the genetic code of different types of macrophages to find non-immune genes highly active in muscularis macrophages, identifying bone morphogenetic protein 2. BMP2 is one of a family of proteins thought to control organ development.

Blocking the effect of BMP2 mirrored the effects of the macrophage removal, confirming that the protein is used for regulation of intestinal movements. The BMP2 is used by neighboring nerve cells, intestinal neurons, which in turn secrete a protein called colony stimulatory factor 1 (CSF1) that supports macrophages.

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Immune Cell's Role in Intestinal Movement Could Lead to Better Understanding of IBS

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Newswise Researchers at the National Institutes of Health have developed a technique that will speed up the production of stem-cell derived tissues. The method simultaneously measures the expression of multiple genes, allowing scientists to quickly characterize cells according to their function and stage of development. The technique will help the researchers in their efforts to use patients skin cells to regenerate retinal pigment epithelium (RPE)a tissue in the back of the eye that is affected in several blinding eye diseases. It will also help the scientists search for drugs for personalized treatments.

Progress in stem cell-based therapies has been limited by our capacity to authenticate cells and tissues, said Kapil Bharti, Ph.D., a Stadtman Investigator in the Unit on Ocular and Stem Cell Translational Research at the National Eye Institute (NEI), a part of NIH. This assay expands that capacity and streamlines the process.

The assay was described in a recent issue of Stem Cells Translational Medicine.

The RPE is a single layer of cells that lies adjacent to the retina, where the light-sensitive photoreceptors commonly called rods and cones are located. The RPE supports photoreceptor function. Several diseases cause the RPE to break down, which in turn leads to the loss of photoreceptors and vision.

The stem cells Dr. Bharti is using to make RPE are induced pluripotent (iPS) stem cells, which are produced by reverting mature cells to an immature state, akin to embryonic stem cells. iPS cells can be derived from a patients skin or blood cells, coaxed into other cell types (such as neurons or muscle), and in theory, re-implanted without causing immune rejection.

To verify the identity of RPE made from iPS cells, scientists use microscopy to ensure the tissue looks like RPE and physiological assays to ensure the tissue behaves like RPE. They also use a technique called quantitative RT-PCR to measure the expression of genes that indicate ongoing cell development and function. For example, expression of the gene SOX2 is much higher in iPS cells than mature RPE.

But quantitative RT-PCR only permits the simultaneous measurement of a few genes per sample. Dr. Bharti teamed up with Marc Ferrer, Ph.D., of NIHs National Center for Advancing Translational Sciences (NCATS) to develop a multiplex assaya method for simultaneously measuring multiple genes per RPE sample in a highly automated fashion. The assay is based on a commercially available platform from the biotech company Affymetrix. In the assay, tiny snippets of DNA tethered to beads are used to capture RNA moleculescreated when genes are expressed by cells in the RPE sample. Once captured, the RNA from distinct genes is labeled with a fluorescent tag.

Starting with cells from a skin biopsy, the researchers generated iPS-derived RPE and then measured the expression of eight genes that are markers of development, function, and disease. They measured RNA levels of each gene one at a time using quantitative RT-PCR and then all genes simultaneously using the multiplex assay. When compared, the results correlated.

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Gene Profiling Technique to Accelerate Stem Cell Therapies for Eye Diseases