title: [Gene therapy for hereditary eye diseases: where are we?].
abstract: Recently, preliminary results of three clinical gene therapy trials for early onset retinitis pigmentosa--Leber congenital amaurosis--suggested that treating this degenerative retinal disease by gene transfection can be safe and efficient to restore a visual function. The definitive validation of this therapeutic approach depends on the long-term results. The forthcoming availability of gene therapy in ophthalmology prompts the implementation: of 1) recruitment, 2) phenotyping and genotyping of affected patients, 3) and creation of a hereditary retinopathy registry.
authors.affiliation: Unité de phénotypage et thérapie moléculaire, Hôpital ophtalmique universitaire Jules-Gonin, 1004 Lausanne. h.tran@ucl.ac.uk
authors.forename: Hoai
authors.initials: H
authors.lastname: Viet Tran
authors.forename: Daniel F
authors.initials: DF
authors.lastname: Schorderet
authors.forename: Corinne
authors.initials: C
authors.lastname: Kostic
authors.forename: Francis L
authors.initials: FL
authors.lastname: Munier
authors.forename: Yvan
authors.initials: Y
authors.lastname: Arsenijevic
mesh_terms: D015785:Eye Diseases, Hereditary / Q000628:therapy*; D015316:Genetic Therapy; D006801:Humans