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Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

1 Dec 2009

Abstract excerpt

The safety and efficacy of gene therapy for inherited retinal diseases is being tested in humans affected with Leber's congenital amaurosis (LCA), an autosomal recessive blinding disease. Three independent studies have provided evidence that the subretinal administration of adeno-associated viral (AAV) vectors encoding RPE65 in patients affected with LCA2 due to mutations in the RPE65 gene, is safe and, in some...

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PMID: 19953081
DOI: 10.1038/mt.2009.277

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Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector AdministrationPMID 19953081Read ↗

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The Status of RPE65 Gene Therapy Trials: Safety and Efficacy.Semantic proximity Read ↗

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