ArticleScience translational medicine2015
Plasticity of the human visual system after retinal gene therapy in patients with Leber's congenital amaurosis.
Article in Science translational medicine, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 44 papers, 1 of them a synthesis that pooled it.
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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
44 citing papers in PubMed, 1 synthesis or guideline pooled it, 63 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
- Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.Lancet (London, England) · 2017Trial
- Clinical and genetic features ofFrontiers in medicine · 2025Article
- Partial rescue of the full-field electroretinogram in patients with RPE65-related retinal dystrophy following gene augmentation therapy with voretigene neparvovec-rzyl.Documenta ophthalmologica. Advances in ophthalmology · 2024Article
- Central visual pathways affected by degenerative retinal disease before and after gene therapy.Brain : a journal of neurology · 2024Article
- Voretigene neparvovec for inherited retinal dystrophy due to RPE65 mutations: a scoping review of eligibility and treatment challenges from clinical trials to real practice.Eye (London, England) · 2024Article
- Longitudinal stability of individual brain plasticity patterns in blindness.Proceedings of the National Academy of Sciences of the United States of America · 2024Article
- Longitudinal stability of individual brain plasticity patterns in blindness.bioRxiv : the preprint server for biology · 2023Article
- Achromatopsia-Visual Cortex Stability and Plasticity in the Absence of Functional Cones.Investigative ophthalmology & visual science · 2023Article
- Stages, pathogenesis, clinical management and advancements in therapies of age-related macular degeneration.International ophthalmology · 2023Review
- Lessons Learned from the Development of the First FDA-Approved Gene Therapy Drug, Voretigene Neparvovec-rzyl.Cold Spring Harbor perspectives in medicine · 2023Review
- Dynamic structural remodeling of the human visual system prompted by bilateral retinal gene therapy.Current research in neurobiology · 2023Article
- Brain Gray Matter Atrophy and Functional Connectivity Remodeling in Patients With Chronic LHON.Frontiers in neuroscience · 2022Article
- Neuroplasticity of the Lateral Geniculate Nucleus in Response to Retinal Gene Therapy in a Group of Patients withEye and brain · 2022Article
- Neuroimaging in Leber Hereditary Optic Neuropathy: State-of-the-art and future prospects.NeuroImage. Clinical · 2022Review
- Role of Structural, Metabolic, and Functional MRI in Monitoring Visual System Impairment and Recovery.Journal of magnetic resonance imaging : JMRI · 2021Review
- Gene therapy for inherited retinal diseases.Annals of translational medicine · 2021Review
- RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy.Orphanet journal of rare diseases · 2021Article
- Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- Functional Dynamics of Deafferented Early Visual Cortex in Glaucoma.Frontiers in neuroscience · 2021Article
Corrections and comments
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Authors and funding
12 authors at 5 institutions in 2 countries.
Funding
Abstract
Much of our knowledge of the mechanisms underlying plasticity in the visual cortex in response to visual impairment, vision restoration, and environmental interactions comes from animal studies. We evaluated human brain plasticity in a group of patients with Leber's congenital amaurosis (LCA), who regained vision through gene therapy. Using non-invasive multimodal neuroimaging methods, we demonstrated that reversing blindness with gene therapy promoted long-term structural plasticity in the visual pathways emanating from the treated retina of LCA patients. The data revealed improvements and normalization along the visual fibers corresponding to the site of retinal injection of the gene therapy vector carrying the therapeutic gene in the treated eye compared to the visual pathway for the untreated eye of LCA patients. After gene therapy, the primary visual pathways (for example, geniculostriate fibers) in the treated retina were similar to those of sighted control subjects, whereas the primary visual pathways of the untreated retina continued to deteriorate. Our results suggest that visual experience, enhanced by gene therapy, may be responsible for the reorganization and maturation of synaptic connectivity in the visual pathways of the treated eye in LCA patients. The interactions between the eye and the brain enabled improved and sustained long-term visual function in patients with LCA after gene therapy.
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Registered trials
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.