“Latest Stem Cells News”

A team of researchers at the UC Davis Institute for Regenerative Cures has developed a technique for using stem cells to deliver therapy that specifically targets the genetic abnormality found in Huntington’s disease, a hereditary brain disorder that causes progressive uncontrolled movements, dementia and death. The findings, now available online in the journal Molecular and Cellular Neuroscience, suggest a promising approach that might block the disease from advancing.

“For the first time, we have been able to successfully deliver inhibitory RNA sequences from stem cells directly into neurons, significantly decreasing the synthesis of the abnormal huntingtin protein,” said Jan A. Nolta, principal investigator of the study and director of the UC Davis stem cell program and the UC Davis Institute for Regenerative Cures. “Our team has made a breakthrough that gives families affected by this disease hope that genetic therapy may one day become a reality.”

Huntington’s disease can be managed with medications, but currently there are no treatments for the physical, mental and behavioral decline of its victims. Nolta and other experts think the best chance to halt the disease’s progression will be to reduce or eliminate the mutant huntingtin (htt) protein found in the neurons of those with the disease. RNA interference (RNAi) technology has been shown to be highly effective at reducing htt protein levels and reversing disease symptoms in mouse models.

“Our challenge with RNA interference technology is to figure out how to deliver it into the human brain in a sustained, safe and effective manner,” said Nolta, whose lab recently received funding from the California Institute for Regenerative Medicine to develop an RNAi delivery system for Huntington’s disease. “We’re exploring how to use human stem cells to create RNAi production factories within the brain.”

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An agreement on funding stem cell research is closer than previously thought. A change in direction on financing stem cell research by US President Barack Obama has excited the American scientific community, starting with the major universities in California, including the San Francisco State, San Jose’ University, Stanford, and Berkeley. This time it will be the California Institute of Regenerative Medicine (CIRM) to open its wallet, ready to fund grants worth 58 million dollars to groups and researchers studying stem cells.

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The Governing Board of the California Institute for Regenerative Medicine, the State Stem Cell Agency, approved a $25 million award to support the first FDA-approved clinical trial based on cells derived from human embryonic stem cells.

The award to Menlo Park-based Geron, Corp, will support the company’s on-going early phase trial for people with spinal cord injury. This is the first time the agency, which was created by the passage of proposition 71 in 2004, has funded a human clinical trial testing a stem cell-derived therapy.

“Supporting the Geron trial is a landmark step for CIRM,” said Robert Klein, CIRM chairman. “However, we must remember that there will be successes and interim failures as human trials proceed through the refinements necessary to achieve a successful human therapy. We need to be prepared to stand by the heroic patients and the companies as they face these challenges and solve the problems that stand in the way of the recovery of patients from paralysis. When the people of California voted for proposition 71 they did so with the hope of seeing new therapies for disabling diseases like Alzheimer’s disease, Parkinson’s disease, diabetes and other chronic diseases and injuries. By funding this trial, CIRM is taking a major step toward making that hope a reality. ”

The initial phase of the trial will include just a small number of people with recent spinal cord injuries who will receive injections of oligodendrocyte progenitor cells derived from embryonic stem cells into the site of the injury. In animal models, those cells mature into oligodendrocytes, which produce the insulating layer surrounding neurons. The initial phase of the three-year project is designed to test whether the cells are safe. Later phases will include different levels of spinal cord injury and will test increasing doses of the cells. One person has already received injections of the cells at a clinical trial site in Georgia. Stanford University Medical Center is another of the trial locations.

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A gene called SOX2 acts as a stem cell gatekeeper – only cells expressing it have the potential to become neurons.

Early in embryonic development, the neural crest – a transient group of stem cells – gives rise to parts of the nervous system and several other tissues. But little is known about what determines which cells become neurons and which become other cell types. A team led by Dr. Alexey Terskikh at Sanford-Burnham Medical Research Institute (Sanford-Burnham) recently found that expression of a gene called SOX2 maintains the potential for neural crest stem cells to become neurons in the peripheral nervous system, where they interface with muscles and other organs. Their results, published online May 5 by the journal Cell Stem Cell, could help better inform therapies aimed at neurocristopathies, diseases caused by defects in the neural crest or neurons, which include microphthalmia and CHARGE syndrome.

The SOX2 gene encodes a transcription factor, a type of protein that switches other genes on or off. SOX2 is one of two key genes researchers use to generate induced pluripotent stem cells (iPSCs), which are capable of differentiating into all cell types for research and potential therapeutic applications.

“In this study, we looked at SOX2’s role in cells of the peripheral nervous system and discovered that it’s required to sustain multipotency – the ability to differentiate into several cell types in the peripheral nervous system, including neurons and glia,”

…explained Dr. Terskikh, assistant professor in Sanford-Burnham’s Del E. Webb Neuroscience, Aging and Stem Cell Research Center.

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Stanford University’s Faculty Senate today approved the creation of what officials believe is the first PhD program devoted solely to stem cell science in the nation and, perhaps, the world. The new doctoral program in stem cell biology and regenerative medicine is also the first interdisciplinary doctoral program created by the School of Medicine in recent years.

School officials say the fact that the university is taking the rare step of creating a new doctoral program acknowledges the growing importance of stem cell research in the realm of biomedical science. The senate’s initial approval of the program extends for five years.

“Stem cell biology is a distinct discipline that requires unique skills and includes a scope of knowledge and a skill set that is not covered by other disciplines,” said Renee Reijo Pera, PhD, professor of obstetrics and gynecology and director of the new PhD program.

Program leaders note that Stanford is among a small number of U.S. universities that have the necessary ingredients to create a doctoral program teaching the full range of stem cell science. They add that although a few other schools have recently established PhD programs involving stem cell biology, Stanford is the first to create a free-standing doctoral program dedicated solely to stem cell biology and regenerative medicine.

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Abundant precursor cells can become many types of neurons without introducing tumor risk

In a paper published in the April 25 early online edition of the Proceedings of the National Academy of Sciences, researchers at the University of California, San Diego School of Medicine, the Gladstone Institutes in San Francisco and colleagues report a game-changing advance in stem cell science: the creation of long-term, self-renewing, primitive neural precursor cells from human embryonic stem cells (hESCs) that can be directed to become many types of neuron without increased risk of tumor formation.

“It’s a big step forward,” said Kang Zhang, MD, PhD, professor of ophthalmology and human genetics at Shiley Eye Center and director of the Institute for Genomic Medicine, both at UC San Diego. “It means we can generate stable, renewable neural stem cells or downstream products quickly, in great quantities and in a clinical grade – millions in less than a week – that can be used for clinical trials and, eventually, for clinical treatments. Until now, that has not been possible.”

Human embryonic stem cells hold great promise in regenerative medicine due to their ability to become any kind of cell needed to repair and restore damaged tissues. But the potential of hESCs has been constrained by a number of practical problems, not least among them the difficulty of growing sufficient quantities of stable, usable cells and the risk that some of these cells might form tumors.

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