ArticleFrontiers in neurology2021
Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A
Registry-linked trialAbstract read
In one paragraph
Article in Frontiers in neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05555784 (Evaluation of Impact of Disease and Visual Disability on Quality of Life and Loss of Independence of Patients Living in France With Leber's Hereditary Optic Neuropathy), which is not on this map. Cited by 40 papers.
0numbers the graph read from it
0cells of the map it votes in
40citing papers in PubMed
–field-weighted citation impact
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Evaluation of Impact of Disease and Visual Disability on Quality of Life and Loss of Independence of Patients Living in France With Leber's Hereditary Optic Neuropathy (LHON) Through Qualitative and Quantitative Data Collection
Who cites it
40 citing papers in PubMed.
- Predictors of Final Visual Outcome in Patients With Leber Hereditary Optic Neuropathy Treated With Lenadogene Nolparvovec Gene Therapy.Investigative ophthalmology & visual science · 2025Trial
- Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy.JAMA ophthalmology · 2025Trial
- Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy.Brain : a journal of neurology · 2023Trial
- Advances in Primary Mitochondrial Diseases: Diagnosis, Natural History Studies and Clinical Trials.Genes · 2026Review
- Modalities of vision restoration in optic neuropathies and retinal disease.Progress in retinal and eye research · 2026Review
- Emerging therapeutic strategies for mitochondrial DNA-related diseases.Cell reports. Medicine · 2026Review
- Advanced therapies for inherited optic neuropathies.Eye (London, England) · 2026Review
- Ten-year natural history of visual function in Japanese patients with Leber hereditary optic neuropathy: A retrospective cohort study.PloS one · 2026Observational
- Mitochondrial transfer and mesenchymal stem cells in ophthalmology: current evidence and therapeutic implications.Experimental biology and medicine (Maywood, N.J.) · 2026Review
- Advances in gene therapy for mitochondrial genetic disorders: current status and clinical implementation challenges.Journal of translational medicine · 2025Review
- Preclinical Assessment of Mitochondrial-Targeted ND4 Gene Therapy for Leber Hereditary Optic Neuropathy.Investigative ophthalmology & visual science · 2025Article
- Therapies for Mitochondrial Disease: Past, Present, and Future.Journal of inherited metabolic disease · 2025Review
- Article
- Engineered mitochondria in diseases: mechanisms, strategies, and applications.Signal transduction and targeted therapy · 2025Review
- Mitochondria in Retinal Ganglion Cells: Unraveling the Metabolic Nexus and Oxidative Stress.International journal of molecular sciences · 2024Review
- Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial.Cell reports. Medicine · 2024Article
- Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care.Biotech (Basel (Switzerland)) · 2024Review
- Review
- 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
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
21 authors.
Nancy J NewmanDepartments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA, United States.
Patrick Yu-Wai-ManCambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Department of Clinical Neurosciences, University of Cambridge, Cambridge, United Kingdom.
Valerio CarelliIstituto di Ricovero e Cura a Carattere Scientifico Istituto delle Scienze Neurologiche di Bologna, Unitá Operativa Compless Clinica Neurologica, Bologna, Italy.
Valerie BiousseDepartments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA, United States.
Mark L MosterDepartments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, United States.
Catherine Vignal-ClermontDepartment of Neuro Ophthalmology and Emergencies, A. de Rothschild Foundation Hospital, Paris, France.
Robert C SergottDepartments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, United States.
Thomas KlopstockDepartment of Neurology, Friedrich-Baur-Institute, University Hospital, Ludwig-Maximilians-University Munich, Munich, Germany.
Alfredo A SadunDoheny Eye Institute, School of Medicine, University of California, Los Angeles, Los Angeles, CA, United States.
Jean-François GirmensCentre d'investigation Clinique, Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, Paris, France.
Chiara La MorgiaIstituto di Ricovero e Cura a Carattere Scientifico Istituto delle Scienze Neurologiche di Bologna, Unitá Operativa Compless Clinica Neurologica, Bologna, Italy.
Adam A DeBuskDepartments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, United States.
Neringa JurkuteMoorfields Eye Hospital National Health Service Foundation Trust, London, United Kingdom.
Claudia PriglingerDepartment of Ophthalmology, University Hospital, Ludwig-Maximilians-University Munich, Munich, Germany.
Rustum KaranjiaDoheny Eye Institute, School of Medicine, University of California, Los Angeles, Los Angeles, CA, United States.
Constant JosseeXYSTAT, Data Management and Statistic, Malakoff, France.
Julie SalzmannMedical and Regulatory Consulting, Paris, France.
François MontestruceXYSTAT, Data Management and Statistic, Malakoff, France.
Michel RouxGenSight Biologics, Paris, France.
Magali TaielGenSight Biologics, Paris, France.
José-Alain SahelSorbonne Université, INSERM, CNRS, Institut de la Vision, Paris, France.
Funding
P30-Core Grant for Vision Research Core CP30EY006360 · NEI · EMORY UNIVERSITY · PI BOATRIGHT, JEFFREY H · 1986 to 2025
$15.3MMedical Research Council G0701386Medical Research Council G1002570NEI NIH HHS P30 EY006360
Abstract
PubMed holds no abstract for this paper.
Indexed as
gene therapyLeber hereditary optic neuropathynatural historyND4visual acuity
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY
Read underepoch 390
Registered trials
NCT05555784ClinicalTrials.gov
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.