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Archive for the Gene Therapy Category

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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Holiday Giving Means Doubling Your Gift

Allison Corona videoFor many reasons—not the least of which are holiday-related—December is a month when we all consider ways to give to others. And the spirit of giving has never been more apparent than it is right now at the Foundation Fighting Blindness. Not only is our end-of-year fundraising campaign in full swing; it’s bolstered by the Chairman’s Holiday Match. What that means is, for every dollar someone gives, it’ll be matched by another dollar—thus doubling its value. Give $25, it’s worth $50. Give $50, it’s worth $100. And so on.
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Changing Someone’s Life: A New Video Emphasizes the Need to Support FFB’s Mission

Allison CoronaGrowing up, Allison Corona could see shapes and silhouettes, but not the faces of loved ones clearly enough to know if they were happy or sad. She needed a cane to navigate her schools’ hallways. And in a dark room, or outside at night, she was completely blind. “It was like living a half-life,” Allison, 22, recalls in a new FFB video, “Restoring Sight, One Treatment at a Time.”
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Inspired by Progress in Usher Syndrome Research

Dr. Mark PennesiWhen I first heard about Usher syndrome, the leading cause of combined blindness and deafness, I was 19, fresh out of high school and very privileged for the opportunity to work as a summer student at the Retina Foundation of the Southwest with David Birch, Ph.D. I was quite moved by the patients — their eagerness for any kind of treatment and the fear that they might pass their disease on to other family members. That was almost 20 years ago. Back then, we knew very little about the condition, especially its genetic diversity, and there were no foreseeable therapies.
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How Evolution is Leading to Gene Therapies for More Retinal Diseases

Dr. John FlanneryAt first blush, gene therapy for retinal diseases seems so simple: Inject a tiny drop of liquid containing good copies of a gene to replace the bad, and you’re home free. Vision is saved, and, in some cases, it’s even restored.

But the reality is: Developing gene therapies that are safe, effective and long-lasting is very challenging in our world of genetically diverse retinal degenerations. Scientists have to design a delivery system that gets the genes to the right types of cells across the entire retina, but without affecting other cells.
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