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Archive for the Gene Therapy Category

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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ARVO 2015 Highlight: AMD Gene Therapy Performs Encouragingly in Human Study

an AMD eyeWhile treatments such as Lucentis®, Avastin®, and Eylea® have been saving and restoring vision for people with wet age-related macular degeneration (AMD) over the last several years, they have a significant drawback: The therapies require regular injections into the eye—in some cases, monthly—for the life of the patient.
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ARVO 2015 Highlight: New Research Boosts Prospects for Saving Vision with RdCVF

Dr. SahelAn eye doctor could preserve meaningful vision in people with advanced retinitis pigmentosa (RP) by saving just five percent of their cones, the cells concentrated in the central retina enabling us to read, recognize colors and see in lighted conditions.
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ARVO 2015 Highlight: A Cut-and-Paste Approach to Fixing Retinal-Disease Genes

gene editingI just returned from the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), the world’s largest eye-research conference, held this year in Denver. It attracted more than 11,000 scientists and physicians, including many of the 187 retinal researchers funded by the Foundation. The FFB science team and I worked feverishly to learn as much as possible about the latest news from the retinal-research front. It was truly exhilarating—albeit, at times, overwhelming.
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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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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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