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Article

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

8 Feb 2012

Abstract excerpt

Demonstration of safe and stable reversal of blindness after a single unilateral subretinal injection of a recombinant adeno-associated virus (AAV) carrying the RPE65 gene (AAV2-hRPE65v2) prompted us to determine whether it was possible to obtain additional benefit through a second administration of the AAV vector to the contralateral eye. Readministration of vector to the second eye was carried out in three...

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PMID: 22323828
DOI: 10.1126/scitranslmed.3002865

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