Gene Therapy for Leber Hereditary Optic Neuropathy (LHON): A Systematic Review and Meta-Analysis

Neuro-Ophthalmology 2026 Open access

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Authors: Lama Nasser Alghamdi, Ali Saleh Alsudais, Salma Hamdan Almarwani, Rose A. Alraddadi, Abdulrahman Hamid Alsamadani, Abdulrahman A. Alnamlah, Saif Aljehani, Yousof Fahad Allarakia, Amer M Alghamdi, Daniah Alshowaeir
DOI: 10.1080/01658107.2026.2624440
Cited by: 0
References: 17

Abstract

Background: LHON is a rare mitochondrial disease causing bilateral vision loss, most commonly due to the m.11778G>A mutation. rAAV2/2-ND4 gene therapy is a potential disease-modifying treatment. Methods: Systematic review and meta-analysis of three RCTs (RESCUE, REVERSE, REFLECT) including 175 patients. Results: Gene therapy significantly improved best-corrected visual acuity but did not significantly increase responder rates. Adverse events were mostly mild ocular inflammation, with no treatment-related mortality. Conclusions: rAAV2/2-ND4 is a moderately effective and safe treatment for LHON, though long-term outcomes and predictors of response remain unclear.

Fields of Study & Subjects

Virus-based gene therapy research Retinal Development and Disorders Glaucoma and retinal disorders

Publication details

Journal / source
Neuro-Ophthalmology
Volume
50
Issue
3
Pages
215–221
Publication date
17 February 2026
Language
EN