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Article

Efficacy and Safety of rAAV2-ND4 Treatment for Leber's Hereditary Optic Neuropathy.

Scientific reports - 19 Feb 2016

Wan Xing, Pei Han, Zhao Min-jian, Yang Shuo, Hu Wei-kun, He Heng, Ma Si-qi, Zhang Ge, Dong Xiao-yan, Chen Chen, Wang Dao-wen, Li Bin

Abstract excerpt

Leber's hereditary optic neuropathy (LHON) is a mitochondrially inherited disease leading to blindness. A mitochondrial DNA point mutation at the 11778 nucleotide site of the NADH dehydrogenase subunit 4 (ND4) gene is the most common cause. The aim of this study was to evaluate the efficacy and safety of a recombinant adeno-associated virus 2 (AAV2) carrying ND4 (rAAV2-ND4) in LHON patients carrying the G11778A...

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Identifiers

PMID: 26892229
DOI: 10.1038/srep21587
PMCID: PMC4759604

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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