ArticleGene therapy2021
Sensitization of ON-bipolar cells with ambient light activatable multi-characteristic opsin rescues vision in mice.
Article in Gene therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers, 1 of them a synthesis that pooled it.
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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
25 citing papers in PubMed, 1 synthesis or guideline pooled it, 39 citations in OpenAlex.
- A Systematic Review of Optogenetic Vision Restoration: History, Challenges, and New Inventions from Bench to Bedside.Cold Spring Harbor perspectives in medicine · 2023Pooled it
- Therapeutic Efficacy of Multi-Characteristic Opsin Gene Therapy in a Mouse Model of Stargardt Disease.Bioengineering (Basel, Switzerland) · 2026Article
- Functional methods for evaluating the efficacy of retinal optogenetic therapy for vision restoration.Frontiers in neuroscience · 2026Review
- 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
- A synthetic opsin restores vision in patients with severe retinal degeneration.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
- Evolution of Light-Sensitive Proteins in Optogenetic Approaches for Vision Restoration: A Comprehensive Review.Biomedicines · 2025Review
- Preclinical biodistribution and toxicology assessment of an AAV5-based subretinal modifier gene therapy for retinitis pigmentosa.Frontiers in medicine · 2025Article
- Tackling visual impairment: emerging avenues in ophthalmology.Frontiers in medicine · 2025Review
- Multi-Characteristic Opsin Therapy to Functionalize Retina, Attenuate Retinal Degeneration, and Restore Vision in Mouse Models of Retinitis Pigmentosa.Translational vision science & technology · 2024Article
- Optogenetics and Targeted Gene Therapy for Retinal Diseases: Unravelling the Fundamentals, Applications, and Future Perspectives.Journal of clinical medicine · 2024Review
- Response of heterologously expressed pressure sensor-actuator-modulator macromolecule to external mechanical stress.Heliyon · 2024Article
- The Healthy and Diseased Retina Seen through Neuron-Glia Interactions.International journal of molecular sciences · 2024Review
- A clinically viable approach to restoring visual function using optogenetic gene therapy.Molecular therapy. Methods & clinical development · 2023Article
- An Engineering Platform for Clinical Application of Optogenetic Therapy in Retinal Degenerative Diseases.IEEE journal of translational engineering in health and medicine · 2023Article
- Gene-agnostic approaches to treating inherited retinal degenerations.Frontiers in cell and developmental biology · 2023Review
- Current approaches to vision restoration using optogenetic therapy.Frontiers in cellular neuroscience · 2023Review
- Optogenetic Therapy for Visual Restoration.International journal of molecular sciences · 2022Review
- miR-181a/b downregulation: a mutation-independent therapeutic approach for inherited retinal diseases.EMBO molecular medicine · 2022Article
- Visual function restoration with a highly sensitive and fast Channelrhodopsin in blind mice.Signal transduction and targeted therapy · 2022Article
Corrections and comments
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Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
Gene therapy-based treatment such as optogenetics offers a potentially powerful way to bypass damaged photoreceptors in retinal degenerative diseases and use the remaining retinal cells for functionalization to achieve photosensitivity. However, current approaches of optogenetic treatment rely on opsins that require high intensity light for activation thus adding to the challenge for use as part of a wearable device. Here, we report AAV2 assisted delivery of highly photosensitive multi-characteristic opsin (MCO1) into ON-bipolar cells of mice with retinal degeneration to allow activation by ambient light. Rigorous characterization of delivery efficacy by different doses of AAV2 carrying MCO1 (vMCO1) into targeted cells showed durable expression over 6 months after delivery as measured by reporter expression. The enduring MCO1 expression was correlated with the significantly improved behavioral outcome, that was longitudinally measured by visual water-maze and optomotor assays. The pro/anti-inflammatory cytokine levels in plasma and vitreous humor of the vMCO1-injected group did not change significantly from baseline or control group. Furthermore, biodistribution studies at various time points after injection in animal groups injected with different doses of vMCO1 showed non-detectable vector copies in non-targeted tissues. Immunohistochemistry of vMCO1 transfected retinal tissues showed bipolar specific expression of MCO1 and the absence of immune/inflammatory response. Furthermore, ocular imaging using SD-OCT showed no change in the structural architecture of vMCO1-injected eyes. Induction of ambient light responsiveness to remaining healthy bipolar cells in subjects with retinal degeneration will allow the retinal circuitry to gain visual acuity without requiring an active stimulation device.
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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.