Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy.

doi:10.1001/jamaophthalmol.2024.5375

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Article

Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy.

JAMA ophthalmology - 1 Feb 2025

Yu-Wai-Man Patrick, Newman Nancy J, Biousse Valérie, Carelli Valerio, Moster Mark L, Vignal-Clermont Catherine, Klopstock Thomas, Sadun Alfredo A, Sergott Robert C, Hage Rabih, Degli Esposti Simona, La Morgia Chiara, Priglinger Claudia, Karanja Rustum, Taiel Magali, Sahel José-Alain

Abstract excerpt

Importance: Limited studies have assessed the long-term benefit/risk of gene therapy for Leber hereditary optic neuropathy (LHON). Objective: To determine the safety and efficacy of lenadogene nolparvovec in patients with LHON due to the MT-ND4 gene variant for up to 5 years after administration. Design, Setting, and Participants: The RESCUE and REVERSE Long-Term Follow-up Study (RESTORE), conducted from 2018 to...

Topics

Adolescent
Adult
Female
Humans
Male
Middle Aged
Young Adult
Dependovirus
DNA, Mitochondrial
Follow-Up Studies

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Genetic Therapy

Genetic Vectors

Mutation

NADH Dehydrogenase

Optic Atrophy, Hereditary, Leber

Prospective Studies

Time Factors

Treatment Outcome

Source

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Identifiers

PMID: 39699886
DOI: 10.1001/jamaophthalmol.2024.5375
PMCID: PMC11843360

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