[Gene therapy for hereditary eye diseases: where are we?].
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.
Viet Tran
Hoai
H
Unité de phénotypage et thérapie moléculaire, Hôpital ophtalmique universitaire Jules-Gonin, 1004 Lausanne. h.tran@ucl.ac.uk
Schorderet
Daniel F
DF
Kostic
Corinne
C
Munier
Francis L
FL
Arsenijevic
Yvan
Y
D015785:Eye Diseases, Hereditary / Q000628:therapy*; D015316:Genetic Therapy; D006801:Humans