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Gene therapy may improve visual function in retinal degeneration caused by RPE65 mutations
An adeno-associated virus vector improved one or more measures of visual function in patients with retinal degeneration resulting from RPE65 mutations.Investigators set out to assess the safety of rAAV2-CB-hRPE65, a recombinant adeno-associated virus vector expressing RPE65, a 65-kDa isomerohydrolase produced by retinal pigment epithelium cells. Mutations of RPE65 are believed to result in a severe deficiency of active chromophore and can cause Leber congenital amaurosis and severe early childhood-onset retinal dystrophy.