ArticleClinical & experimental ophthalmology2019
Prognostic factors for visual acuity in patients with Leber's hereditary optic neuropathy after rAAV2-ND4 gene therapy.
Article in Clinical & experimental ophthalmology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 1 of them a synthesis that pooled it.
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Who cites it
20 citing papers in PubMed, 1 synthesis or guideline pooled it, 32 citations in OpenAlex.
- Safety and Efficacy of Adeno-Associated Viral Gene Therapy in Patients With Retinal Degeneration: A Systematic Review and Meta-Analysis.Translational vision science & technology · 2023Pooled it
- Advanced therapies for inherited optic neuropathies.Eye (London, England) · 2026Review
- Comparison of thickness changes in retinal nerve fibre layer in Leber's hereditary optic neuropathy patients with 11778, 14484 and 3460 mutations.Translational pediatrics · 2025Article
- Mitochondria-targeted gene delivery using fluorinated lipid nanoparticles to alleviate Leber's hereditary optic neuropathy.Nature communications · 2025Article
- Preclinical Assessment of Mitochondrial-Targeted ND4 Gene Therapy for Leber Hereditary Optic Neuropathy.Investigative ophthalmology & visual science · 2025Article
- The rAAV2-ND1 gene therapy for Leber hereditary optic neuropathy.Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2025Article
- Therapies for Mitochondrial Disease: Past, Present, and Future.Journal of inherited metabolic disease · 2025Review
- Technological advances in the diagnosis and management of inherited optic neuropathies.Frontiers in neurology · 2025Review
- Clinical follow-up investigation on thickness changes in the peripapillary retinal nerve fibre layer of patients with Leber hereditary optic neuropathy.BMC ophthalmology · 2024Article
- Cell-cell interaction in the pathogenesis of inherited retinal diseases.Frontiers in cell and developmental biology · 2024Review
- Mitochondrially Targeted Gene Therapy Rescues Visual Loss in a Mouse Model of Leber's Hereditary Optic Neuropathy.International journal of molecular sciences · 2023Article
- Current and Future Landscape in Genetic Therapies for Leber Hereditary Optic Neuropathy.Cells · 2023Review
- AAV for Gene Therapy in Ocular Diseases: Progress and Prospects.Research (Washington, D.C.) · 2023Review
- Leber Hereditary Optic Neuropathy: Molecular Pathophysiology and Updates on Gene Therapy.Biomedicines · 2022Review
- Gene therapy for inherited retinal diseases.Annals of translational medicine · 2021Review
- Observation of Peripapillary Choroidal Vascularity in Natural Disease Course and After Gene Therapy for Leber's Hereditary Optic Neuropathy.Frontiers in medicine · 2021Article
- Review
- Emerging model systems and treatment approaches for Leber's hereditary optic neuropathy: Challenges and opportunities.Biochimica et biophysica acta. Molecular basis of disease · 2020Review
- A Single-Arm, Prospective, Exploratory Study to Preliminarily Test Effectiveness and Safety of Skin Electrical Stimulation for Leber Hereditary Optic Neuropathy.Journal of clinical medicine · 2020Article
- Prognostic factors for visual acuity in patients with Leber's hereditary optic neuropathy after rAAV2-ND4 gene therapy.Clinical & experimental ophthalmology · 2019Article
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Authors and funding
7 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
importanceFactors affecting visual acuity prognosis after gene therapy in Leber's hereditary optic neuropathy (LHON) patients with mutation at site 11 778 are unknown.
backgroundTo analyse correlations between visual acuity prognosis and baseline characteristics of LHON after rAAV2-ND4 gene therapy.
designRetrospective study.
participantsFifty-three LHON patients with a mutation at site 11 778.
methodsSingle-eye intravitreal injection of rAAV2-ND4.
main outcome measuresSex, onset age, duration of disease, best-corrected visual acuity (BCVA), visual field index (VFI) and mean deviation (MD) were recorded for all patients at baseline. BCVA was recorded at 1- and 3-month follow-up visits after gene therapy. Correlations between BCVA prognosis and baseline characteristics were analysed by univariate analysis. Logistic regression analysis was performed on independent factors affecting BCVA prognosis.
resultsUnivariate analysis showed significant differences in the VFI and MD of the injected eye between BCVA improvement and non-improvement groups after 3 months of treatment, with greater VFI and smaller absolute MD in the BCVA improvement group. Logistic regression showed that VFI and baseline BCVA were independent prognostic factors for visual acuity. The correlation between VFI and MD was statistically significant. CONCLUSIONS AND RELEVANCE: VFI and baseline BCVA were correlated with the visual acuity prognosis of LHON patients receiving gene therapy, with greater baseline VFI and better baseline BCVA predicting better visual acuity prognosis. MD was strongly correlated with VFI and might be correlated with gene therapy prognosis. This finding may form a basis for predicting the efficacy of gene therapy in these patients and guiding subsequent treatment.
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