ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025
A synthetic opsin restores vision in patients with severe retinal degeneration.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed.
- Revisiting retinal and macular degeneration in the genomics era.Nature reviews. Genetics · 2026Review
- Neural Vision Restoration in Ophthalmology.Annals of biomedical engineering · 2026Review
- Modalities of vision restoration in optic neuropathies and retinal disease.Progress in retinal and eye research · 2026Review
- Using Virtual Patients to Predict Perceptual Outcomes for Optogenetic Sight Recovery Technologies.Research square · 2026Article
- Using Virtual Patients to Predict Perceptual Outcomes for Optogenetic Sight Recovery Technologies.bioRxiv : the preprint server for biology · 2026Article
- [Clinical endpoints and translational challenges].Die Ophthalmologie · 2026Review
- Functional methods for evaluating the efficacy of retinal optogenetic therapy for vision restoration.Frontiers in neuroscience · 2026Review
- Bionic vision technologies: progress and perspectives on retinal prostheses and optogenetics for the treatment of advanced retinal degeneration.Frontiers in medical technology · 2026Review
- Perceptual learning of prosthetic vision using video game training.Journal of vision · 2025Article
- Review
- Safety and efficacy of MCO-010 optogenetic therapy in patients with Stargardt disease in USA (STARLIGHT): an open-label multi-center Ph2 trial.EClinicalMedicine · 2025Article
- Perspectives and open questions in vision restoration with vMCO-010.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Response: Promise and open questions of optogenetic vision restoration by MCO.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Regulatory aspects of optogenetic research and therapy for retinitis pigmentosa under EU law.Frontiers in medical technology · 2025Review
- Sensing a rainbow of colors: algal photoreceptors.Frontiers in plant science · 2025Review
- Preclinical biodistribution and toxicology assessment of an AAV5-based subretinal modifier gene therapy for retinitis pigmentosa.Frontiers in medicine · 2025Article
Corrections and comments
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Inherited retinal degenerations are the leading cause of blindness worldwide, and, in advanced stages, cell loss makes gene replacement ineffective. Optogenetics offers a therapeutic opportunity to restore vision by photo-sensitizing remaining retinal neurons. However, current opsins are kinetically slow, partially activated in ambient light, unresponsive to different light colors, and target low-resolution retinal cell circuits. To overcome these limits, we engineered a synthopsin made of three selectively mutated non-mammalian proteins to achieve a broadband multi-characteristic opsin. The synthopsin was packaged into an optimized AAV2 gene-therapy vector that targets human retinal bipolar cells. In an investigator-initiated, open-label study, four blind retinitis pigmentosa patients with ABCA4 variants received a single intravitreal gene-therapy injection. Noninvasive imaging confirmed retinal gene expression via a fluorescent reporter protein. Patients showed improvement in vision, shape discrimination, and mobility through 52 weeks. There were no significant safety issues despite what is likely one of the most synthetic, non-mammalian proteins ever expressed in a human. This is the first report of a gene monotherapy that can restore vision in blind patients in a mutation-independent manner utilizing an optogenetics technology platform.
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Registered trials
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