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.
This activity is supported by an educational grant from Alcon Research, LTD., Allergan, Inc., Bausch + Lomb, and Shire.
Highlights from the Hawaiian Eye/Retina meeting
This activity is supported by an educational grant from Bausch + Lomb.
This activity is supported by educational grants from Allergan, Inc.; Genentech, Inc.; and Regeneron Pharmaceuticals, Inc.
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