ArticleGraefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie2025
The rAAV2-ND1 gene therapy for Leber hereditary optic neuropathy.
Article in Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
3 citing papers in PubMed.
- Pharmacological Depletion of Retinal Mononuclear Phagocytes Is Neuroprotective in a Mouse Model of Mitochondrial Optic Neuropathy.Investigative ophthalmology & visual science · 2026Article
- Visual Neurorestoration: An Expert Review of Current Strategies for Restoring Vision in Humans.Brain sciences · 2025Review
- Isolated and Syndromic Genetic Optic Neuropathies: A Review of Genetic and Phenotypic Heterogeneity.International journal of molecular sciences · 2025Review
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Authors and funding
7 authors.
Funding
Abstract
purposeNo effective treatment for leber hereditary optic neuropathy (LHON) caused by ND1 mutation is available.This study evaluated the safety and efficacy of a single unilateral intravitreal injection rAAV2-ND1 in various doses for the treatment of LHON.
methodsTwelve patients with LHON (ND1 mutation) in two groups with six participants each.The low-dose group received injection of rAAV2-ND1 in a dose of 1.5 × 10
resultsIn total,11 mild eye-related AEs occurred in the participants in both groups, and short-term drug treatment returned to normal.The difference was statistically significant in BCVA of the injected eyes in the low-dose group between 12 months after treatment and at baseline.The differences in BCVA of the uninjected eyes in the high-dose group between baseline and 3 months or 6 months after treatment were statistically significant.At 12 months after treatment, the rate of improvement in BCVA for the injected eyes in the low-dose groups was 66.7% (4/6),while BCVA for the uninjected eyes in the high-dose groups was 50.0% (3/6),and patients in both groups had binocular VF (VFI, MD) and RNFL thicknesses that did not significantly differ from baseline.
conclusionPreliminary clinical evidence shows that rAAV2-ND1 ophthalmic injection is a safe and effective treatment for LHON due to ND1 mutation.
trial registrationTrial registration number: ChiCTR2000041574, Date:12/29/2020.
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