Gene Therapy Versus Retinal Degeneration

Via EurekAlert: "Researchers have used gene therapy to restore useful vision to mice with degeneration of the light-sensing retinal rods and cones, a common cause of human blindness. ... This is a proof of principle that someday we may be able to repair blindness in people with conditions like retinitis pigmentosa and macular degeneration. There are several limitations we need to overcome before we can begin clinical trials, but I'm optimistic that this work may someday make a big difference for people who otherwise would have no vision at all. ... The study was designed to investigate the effect of expressing the light-sensitive protein melanopsin in retinal ganglion cells. These specialized neurons receive light signals from the rods and cones and carry those signals into the brain via the optic nerve, which is formed from the cells' axons. Melanopsin is usually produced in a subset of cells that are involved with establishing circadian rhythms but not with vision. The [researchers] used the standard viral vector adeno-associated virus to deliver the gene encoding melanopsin throughout the retinas of mice whose rod and cone photoreceptors had degenerated from lack of a crucial protein."



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