Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

G. Jacobson, Samuel

doi:10.1001/archophthalmol.2011.298

Public

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

Published in Archives of Ophthalmology • Sep 13, 2011

Authors:

Samuel G. Jacobson

Abstract

OBJECTIVE: To determine the safety and efficacy of subretinal gene therapy in the RPE65 form of Leber congenital amaurosis using recombinant adeno-associated virus 2 (rAAV2) carrying the RPE65 gene. DESIGN: Open-label, dose-escalation phase I study of 15 patients (range, 11-30 years of age) evaluate...

Subject

Visual acuity

Medicine

RPE65

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