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Article

Gene Therapy for Leber Hereditary Optic Neuropathy: Initial Results.

Ophthalmology - 1 Mar 2016

Feuer William J, Schiffman Joyce C, Davis Janet L, Porciatti Vittorio, Gonzalez Phillip, Koilkonda Rajeshwari D, Yuan Huijun, Lalwani Anil, Lam Byron L, Guy John

Abstract excerpt

PURPOSE: Leber hereditary optic neuropathy (LHON) is a disorder characterized by severe and rapidly progressive visual loss when caused by a mutation in the mitochondrial gene encoding NADH:ubiquinone oxidoreductase subunit 4 (ND4). We have initiated a gene therapy trial to determine the safety and tolerability of escalated doses of an adeno-associated virus vector (AAV) expressing a normal ND4 complementary DNA...

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Identifiers

PMID: 26606867
DOI: 10.1016/j.ophtha.2015.10.025
PMCID: PMC5287094

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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