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Archive for the AMD Category

FFB Goes Live to Help Save Vision

Save Your Vision Live!It’s no mystery that the Foundation Fighting Blindness’ core mission is funding research for treatments and cures of retinal diseases, and that we’re making significant progress. A recent influx of clinical, or human, trials of drug, gene and stem-cell therapies is proof. But another key FFB focus is informing the masses about retinal diseases through our public health-education program.
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ARVO 2014: Three Promising CEP290 Gene Therapy Alternatives

Renee Ryalls explains the dual-AAV gene therapy she's developing.While gene therapies for retinal degenerative diseases are making groundbreaking strides in both human and laboratory studies, the most widely and successfully used human-engineered virus for delivering replacement genes to retinal cells — the adeno-associated virus, or AAV — has one significant limitation. It can’t deliver relatively large genes, namely those larger than about 4.5 or 5 kilobases (kb). (Bases are the building blocks of a gene, and its size is expressed in kilobases.)
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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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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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International AMD Awareness Week

A senior coupleSeeing as it’s the leading cause of blindness in people 55 and older, and affects 10 million people in the United States and three times that amount worldwide, it only makes sense that age-related macular degeneration would get its own week. International AMD Awareness Week, which began Sept. 15 and runs through the 22nd, is the brainchild of AMD Alliance International, of which FFB is a member. And it’s imperative that everyone, not just those currently affected, take note.
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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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RetNet: A Web-Based Guide to Every Retinal Disease Gene Known to Man

Dr. Stephen DaigerIt isn’t as addictive as posting your status to Facebook. Nor is it as entertaining as cat blooper videos on YouTube. And it won’t let you send 140-character rants about your political views through cyberspace. But if you are in any way connected to the world of retinal research, you’ll want to check out RetNet, the online catalogue of every known retinal degenerative disease gene. After you do, Steve Daiger, Ph.D., RetNet’s creator, will no doubt become your BFF.
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Update on Six Gene Therapy Clinical Trials Presented at VISIONS 2013

Dr Shannon BoyeWhether you’re a researcher or affected by a retinal disease, hearing the latest news from the clinical-trial front lines is a great perk of FFB’s annual conference. On the last day of VISIONS 2013, I had the privilege of moderating a compelling update on six gene-therapy clinical trials provided by Shannon Boye, Ph.D., a gene therapy developer at the University of Florida, and Michel Michaelides, M.D., a clinician-researcher at Moorfields Eye Hospital in the United Kingdom. All of these emerging treatments, given in recaps below, were made possible by Foundation funding.
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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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