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Article

Gene therapy rescues cone function in congenital achromatopsia

8 Apr 2010

Abstract excerpt

The successful restoration of visual function with recombinant adeno-associated virus (rAAV)-mediated gene replacement therapy in animals and humans with an inherited disease of the retinal pigment epithelium has ushered in a new era of retinal therapeutics. For many retinal disorders, however, targeting of therapeutic vectors to mutant rods and/or cones will be required. In this study, the primary cone...

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Identifiers

PMID: 20378608
DOI: 10.1093/hmg/ddq136

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