Neuro-ophthalmology (Aeolus Press) · 2026
Gene Therapy for Leber Hereditary Optic Neuropathy (LHON): A Systematic Review and Meta-Analysis.
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Alghamdi Lama Nasser, Alsudais Ali Saleh, Almarwani Salma Hamdan, Alraddadi Rose Abdullah, Alsamadani Abdulrahman Hamid, Alnamlah Abdulrahman Ahmed, Aljehani Saif Kamal, Allarakia Yousof Fahad, Alghamdi Amer M, Alshowaeir Daniah
Аннотация
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.
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.