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Posts tagged adeno-associated virus

Optogenetic Therapy Takes First Step Forward in Clinical Trial

Retrosense logoRetroSense Therapeutics has reported that three participants have received injections of its potential optogenetic therapy, known as RST-01, in a Phase I/II clinical trial. The patients were given the lowest dose of RST-01, and no adverse ocular events were observed. Furthermore, the treatment showed some biological activity, though RetroSense did not provide details about what that activity was or what it meant.

More information on safety and efficacy will likely be reported about the RetroSense trial after more trial participants have been observed over a longer period of time, and after discussions with the U.S. Food and Drug Administration. Continue Reading…

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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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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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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