Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy

Richard G. Weleber, Mark E. Pennesi, David J. Wilson, Shalesh Kaushal, Laura R. Erker, Lauren Jensen, Maureen T. McBride, Terence R. Flotte, Margaret Humphries, Roberto Calcedo, William W. Hauswirth, Jeffrey D. Chulay, J. Timothy Stout

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