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Article

Development of Two In Vitro ND1-LHON Models for Evaluating Gene Therapy Efficacy.

Investigative ophthalmology & visual science - 1 Oct 2025

Li Xin, Yuan Jun, Chen Zhang, Zhang Yue, Liu Yuefeng, Zhang Yong

Abstract excerpt

Purpose: The purpose of this study was to address the lack of effective treatments for NADH-ubiquinone oxidoreductase chain 1 (ND1)-related Leber hereditary optic neuropathy (LHON), this study aimed to (1) establish in vitro models mimicking mitochondrial dysfunction in LHON and (2) evaluate the therapeutic potential of recombinant adeno-associated virus (AAV)-mediated ND1 gene therapy (rAAV-ND1). Methods: Two in...

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Identifiers

PMID: 41129129
DOI: 10.1167/iovs.66.13.34
PMCID: PMC12553470

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