title: Gene therapy for Leber congenital amaurosis.
abstract: Recent success in delivering vision to a canine model of a severe, early-onset blinding disease, Leber congenital amaurosis (LCA) (Acland et al 2001) demonstrates that adeno-associated virus serotype 2 (AAV2) is capable of delivering a corrective gene to the target retinal cells. Results of these studies indicate long-term rescue of vision as assessed by psychophysical, behavioural and molecular biological studies. Preliminary results of studies in progress are described and the implications of these results with respect to developing human clinical trials for LCA and for other retinal degenerative diseases are discussed.
authors.affiliation: FM Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, 310 Stellar-Chance Labs, 422 Curie Boulevard, Philadelphia, PA 19104-2689, USA.
authors.forename: Jean
authors.initials: J
authors.lastname: Bennett
mesh_terms: D000818:Animals; D001766:Blindness / Q000235:genetics / Q000628:therapy*; D002352:Carrier Proteins; D000229:Dependovirus; D004285:Dogs; D005136:Eye Proteins; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D051379:Mice; D029242:Optic Atrophy, Hereditary, Leber / Q000235:genetics / Q000628:therapy*; D011506:Proteins / Q000235:genetics / Q000378:metabolism; D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase
chemical_list: D002352:Carrier Proteins; D005136:Eye Proteins; D011506:Proteins; D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase