Preliminary Safety and Tolerability Results Of A Recombinant Adeno-Associated Viral Vector Serotype 2 (Raav2/2) Containing the Human Wild-Type Mitochondrial NADH Dehydrogenase 4 (ND4) Gene, in Patients with Leber Hereditary Optic Neuropathy Due to the G11778A Mitochondrial DNA Mutation (Video)

Abstract

Our goal is to report the results of a first-in-man safety trial of gene therapy in patients with Leber Hereditary Optic Neuropathy (LHON)