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Archive for the Stem Cell Category

ARVO 2015 Highlight: The National Eye Institute Invests $4 Million in Audacious-Goals Research

future of retinal researchThe National Eye Institute’s (NEI) establishment of its Audacious Goals in 2013 was a watershed moment in the drive to cure blinding retinal diseases. The mission of the program—to regenerate the neurons and neural connections in the eye and visual system—is synonymous with the Foundation’s mission to eradicate retinal diseases. Most important, it means that the NEI is making significant investments in research that will benefit people with retinal conditions, such as retinitis pigmentosa, Usher syndrome, Stargardt disease, and age-related macular degeneration.
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Need-to-Know Information about Clinical Trials

clinical trial patientWith about 15 clinical trials underway for inherited retinal diseases, and several more poised to begin in a few years, patients are eager to sign up for access to potential vision-saving therapies.
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A Stem-Cells Video for Kids (and Newbies of All Ages)

Animated videoGiven that inherited retinal diseases are often diagnosed in childhood, it’s only fitting that kids get the opportunity to learn about the promising research and emerging treatments under development to save and restore their vision. Of course, the biology and genetics can be difficult to understand for even an educated adult, so when something kid-friendly is produced, especially a video, we are eager to share it.
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The Top Research Advancements of 2014: How Fast Can We Go?

lab photoAs we approach 2015, it’s inspiring to look back on 2014 and recount the numerous advancements we’ve made in developing vision-saving treatments and cures. When I joined the Foundation nearly a decade ago, virtually nothing was in a human study. We were curing lots of blind mice, and clinical trials seemed elusive. But, today, more than a dozen promising therapies are being evaluated in people, and at least a dozen more clinical trials are expected to begin in the next few years.
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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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