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Archive for the Stem Cell Category

UCI Stem Cell Pioneer Poised to Launch Clinical Trial for RP Patients

Dr. Henry Klassen, a Foundation-funded researcherHenry Klassen, M.D., Ph.D., is only about a year from launching a clinical trial for a stem cell treatment to rescue vision in people with retinitis pigmentosa (RP) and, potentially, other retinal diseases. At the Foundation’s recent Orange County Innovation Symposium, the University of California (UC), Irvine clinician-researcher reported that he and his colleagues are in the midst of final lab studies with patient-grade cells. Once finished, they’ll request authorization from the U.S. Food and Drug Administration to begin their human study.
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Stem Cell Researcher Requests Retraction of Papers on Purported Breakthrough

Haruko ObokataSometimes research, especially early research, isn’t all it’s initially cracked up to be.

Such is the case with a purported advancement I blogged about last month in which researchers reported that soaking cells in a mildly acidic solution might be a safer and easier way to make stem cells. Investigators from the Riken Institute had just published two papers in the prestigious journal Nature detailing the promising approach.
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Stem Cells Derived from Patient’s Skin Provide Insights into AMD

Dr. Stephen TsangInduced pluripotent stem cells (iPSC) — stem cells derived by genetically tweaking a small sample of a person’s skin or blood — are again demonstrating their power for helping researchers fight retinal diseases.

In this latest development, Stephen Tsang, M.D., Ph.D., a Foundation-funded researcher at Columbia University, used them to create a human model of age-related macular degeneration (AMD). The advancement not only gives us a better understanding of how AMD occurs; it provides a new, and potentially better, platform for testing vision-saving therapies. Results of the study were published in Human Molecular Genetics.

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When Research Offers More Than Just Hope

John and Pam CorneilleI’ve lost a lot of my sight to retinitis pigmentosa, or RP, and as Dr. Edwin Stone is conducting exciting stem cell research focused on the disease, I began the process of scheduling an appointment with him last summer. Dr. Stone’s home base is the University of Iowa, which is just a two-and-a-half-hour drive from my house in Illinois. When an appointment date was finally offered, I found it a little odd that it was for New Year’s Eve, but I was eager to go, so I said yes.
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Have Scientists Found a Better Way to Make Stem Cells?

Stem cell cultures, courtesy of the NIHCould soaking a patient’s blood cells in a liquid with the acidity of vinegar be a safer and more effective way to develop stem cells for vision-restoring, retinal-disease treatments? Based on a study recently published in Nature, it might be. The research has a long way to go before it is ready for prime time — i.e., evaluation in humans — but the results thus far are intriguing.
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Stem-Cell Research: Moving at the Speed of Light

Dr. Dennis CleggWhen it comes to designing stem-cell-based treatments for retinal diseases, Dennis Clegg, Ph.D., is one of the go-to researchers. He not only heads his own lab at the University of California at Santa Barbara; he’s the recipient of a CIRM grant for a treatment of age-related macular degeneration, and can turn complicated science into a compelling narrative, as this TED Talk demonstrates.
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Iowa Researchers Use Skin Cells to Gain Insights into Retinitis Pigmentosa

A lab technicianThough retinal researchers have been working with induced pluripotent stem cells (iPSC) for little more than five years, I still find it amazing what they’re doing with them. Scientists are able to take small samples of skin or blood cells from a patient, genetically turn back the clock on those cells, so that they revert back to a stem-cell state, and then coax them forward to become retinal cells. From there, the resulting cells might be used as a transplantation therapy, as a platform for testing potential treatments in a dish and as a resource for learning why retinal diseases cause vision loss and how to prevent it.
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The Foundation’s Center Grants Foster Essential Retinal Research Collaborations

Question: How many researchers does it take to develop a retinal-disease treatment? (No, this isn’t a politically incorrect joke.) The answer is near the end of this article, but no looking ahead!
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Several New Stem Cell Clinical Trials Poised to Begin in Two to Three Years

Microscopic Stem CellsOne of the more exciting aspects of FFB’s recent annual conference, VISIONS 2013, was the news on the clinical development of several emerging stem cell therapies. The field has picked up a lot of steam, with many new human studies of stem-cell treatments anticipated to begin in 2014 and 2015.
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Patient Registries Help Advance Research for Rare Diseases

Image of Keyboard and StethiscopeRare disease research is challenging, because patient information about the conditions is inherently limited. Patient recruitment for clinical trials can be especially difficult, because so few people are affected.

To address these issues, a number of foundations, patient advocacy groups and governmental institutions have launched online registries to collect patient information for use by researchers, doctors and public health experts.
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