1. [Gene therapy for hereditary eye diseases: where are we?]
- Author
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Hoai, Viet Tran, Daniel F, Schorderet, Corinne, Kostic, Francis L, Munier, and Yvan, Arsenijevic
- Subjects
Humans ,Eye Diseases, Hereditary ,Genetic Therapy - 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.
- Published
- 2009