Long-Term Follow-Up After Unilateral Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy: The RESTORE Study.

doi:10.1097/WNO.0000000000001367

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Article

Long-Term Follow-Up After Unilateral Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy: The RESTORE Study.

Journal of neuro-ophthalmology : the official journal of the North American Neuro-Ophthalmology Society - 1 Sept 2021

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

Abstract excerpt

BACKGROUND: RESCUE and REVERSE were 2 Phase 3 clinical trials that assessed the efficacy and safety of intravitreal gene therapy with lenadogene nolparvovec (rAAV2/2-ND4) for the treatment of Leber hereditary optic neuropathy (LHON). RESTORE is the long-term follow-up study of subjects treated in the RESCUE and REVERSE trials. METHODS: In RESCUE and REVERSE, 76 subjects with LHON because of the m.11778 G>A...

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PMID: 34415265
DOI: 10.1097/WNO.0000000000001367
PMCID: PMC8366761

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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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