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Archive for the Gene Therapy Category

Retinal Researchers May Be Looking for You

A patient registers with My Retina Tracker.One of the biggest challenges in overcoming rare retinal diseases is, well, that they’re rare. There’s limited information about the conditions in humans, making it difficult for researchers to understand why they cause blindness and develop vision-saving treatments.
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2015 Top 10 Retinal-Research Advances

Researcher in a labThe Foundation Fighting Blindness’ scientists, donors and volunteers made 2015 an outstanding year in our fight against blindness. As I tabulated the year’s top 10 research advances—all made possible through FFB funding—I realized that eight are for clinical trials of emerging therapies that are launching or underway.
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The Gift of Sight – Matched!

Your donation doubledIt’s holiday time, the giving season, when people not only buy gifts for family, friends and co-workers, but also donate to worthy causes—of which there are many. But, if you’ll permit me, I’d like to make a pitch for the Foundation Fighting Blindness (FFB), which happens to be offering a Holiday Board Match, meaning every donation is doubled.
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A Leap Forward: Spark Therapeutics Seeks FDA Approval for its Vision-Restoring Gene Therapy

Gene therapy recipient Yannick DuweWe’re approaching a critical milestone in the fight against blinding retinal diseases, and it has the potential to tremendously boost and accelerate the advancement of virtually all gene therapies in development for dozens of inherited retinal diseases.

Sometime in 2016, Spark Therapeutics will request marketing approval from the U.S. Food and Drug Administration (FDA) for its landmark gene therapy for retinal conditions caused by mutations in the gene RPE65, namely certain forms of Leber congenital amaurosis and retinitis pigmentosa.
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The Challenge Continues: FFB Chairman Gordon Gund is Interviewed on CNBC

Gordon Gund on CNBC in NantucketGordon Gund, who’s held in extremely high esteem by people inside and outside the Foundation Fighting Blindness (FFB), has been completely blind for decades. He lost his eyesight to a disease called retinitis pigmentosa in his thirties. Not that it slowed him down much. Among other accomplishments, he’s been a financier, venture capitalist, sports-team owner and sculptor. And, of course, he’s a co-founder of FFB and its chairman of the board.
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Gene Therapy Strengthens the Retina’s Connection to the Brain

diagram of brain affected by LCA therapyWhile we often think of the retina as that magical piece of tissue lining the back of the eye that makes vision possible, the brain is also an essential partner in the visual process. When light comes into the eye and is converted to electrical signals, those signals are sent through the optic nerve to the back of the brain, where they are transformed into the images we see. When children are born, pathways between the retina and the brain are in place, and, with increased interaction with the world, they become stronger over time.
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VISIONS 2015 – Dr. José Sahel Receives Foundation’s Most Prestigious Research Honor

Dr. SahelI’ve known Dr. José Sahel for more than a decade, and every time I’m with him, I’m impressed by his humility and graciousness. He’s not much for rhetoric or small talk, but is always polite and insightful. Dr. Sahel is also very soft-spoken, but I think that’s his secret weapon. He forces you to really listen to what he’s saying.
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VISIONS 2015 – Dr. Shannon Boye Receives FFB Award for Excellence in Gene-Therapy Research

Shannon Boye accepting the awardWhen it comes to developing innovative gene therapies for retinal diseases, few researchers are doing more than Shannon Boye, Ph.D., and her laboratory staff of 10 at the University of Florida. In addition to advancing a gene therapy for Leber congenital amaurosis (LCA) caused by GUCY2D mutations toward a human study, Dr. Boye and her team are enhancing gene-delivery systems by making them safer to administer and able to carry larger payloads than current systems.
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Promising Research Highlighted at Meeting of Retinal Gene Therapy Experts

genesWhen it comes to fighting blinding retinal diseases, nothing has been more exciting than the advancement of gene therapies into clinical trials. And, with human studies of gene therapies now underway for Leber congenital amaurosis (LCA, RPE65 mutations), Usher syndrome type 1B, Stargardt disease, retinoschisis, choroideremia and retinitis pigmentosa (MERTK mutations), scientists are gaining new knowledge every day about the best technologies and methodologies for gene-therapy development.
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Sun and Funds: FFB’s Annual Summer Campaign

Summer Challenge web pageWe all know that Memorial Day is not, technically, the first day of summer. But seeing as we like to stretch summer out as much as possible, it only makes sense to kick it off… well… a few weeks early. The same premise is behind FFB’s Summer Challenge to End Blindness campaign, which began Memorial Day weekend and continues through the upcoming season.
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