Gene therapy for hereditary eye diseases: where are we?
Viet Tran, H.; Schorderet, D.F.; Kostic, C.; Munier, F.L.; Arsenijevic, Y.
Revue Medicale Suisse 5(186): 118-123
2009
ISSN/ISBN: 1660-9379 PMID: 19238930 Document Number: 12757
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.
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