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Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations: Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years

JAMA Ophthalmology
Published Online: January 9, 2012 Audio Author Interview 7 min 33 sec
Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations: Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years
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  1. Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

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