ArticleMolecular therapy. Methods & clinical development2023
A clinically viable approach to restoring visual function using optogenetic gene therapy.
Article in Molecular therapy. Methods & clinical development, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 4 registered trials, which are not on this map. Cited by 17 papers.
What it found
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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.
Phase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Intravitreal RST-001 in Patients With Advanced Retinitis Pigmentosa (RP)
A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa
Phase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP in Patients With Retinitis Pigmentosa
A Phase 2b Randomized, Double-Masked, Sham-Controlled, Study to Evaluate the Efficacy and Safety of Intravitreal Injection of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
Who cites it
17 citing papers in PubMed.
- Optogenetic vision restoration: translational barriers and emerging therapeutic strategies.Gene therapy · 2026Review
- Crosstalk-free optical indicator-actuator combinations for bidirectional optogenetics.npj biomedical innovations · 2026Review
- Bionic vision technologies: progress and perspectives on retinal prostheses and optogenetics for the treatment of advanced retinal degeneration.Frontiers in medical technology · 2026Review
- Functional methods for evaluating the efficacy of retinal optogenetic therapy for vision restoration.Frontiers in neuroscience · 2026Review
- The neural thermostat malfunction: revisiting heatstroke through the lens of warm-sensitive neuron dysregulation.Frontiers in cellular neuroscience · 2026Review
- Ion Channel-Targeting Toxins: Structural Mechanisms of Activation, Inhibition, and Therapeutic Potential.Toxins · 2025Review
- Clinical and pharmacovigilance safety evaluation of LUXTURNACutaneous and ocular toxicology · 2025Review
- Marine Derived Strategies Against Neurodegeneration.Marine drugs · 2025Review
- Evolution of Light-Sensitive Proteins in Optogenetic Approaches for Vision Restoration: A Comprehensive Review.Biomedicines · 2025Review
- Regulatory aspects of optogenetic research and therapy for retinitis pigmentosa under EU law.Frontiers in medical technology · 2025Review
- Review
- Optogenetics and Targeted Gene Therapy for Retinal Diseases: Unravelling the Fundamentals, Applications, and Future Perspectives.Journal of clinical medicine · 2024Review
- Theoretical prediction of broadband ambient light optogenetic vision restoration with ChRmine and its mutants.Scientific reports · 2024Article
- Engineered red Opto-mGluR6 Opsins, a red-shifted optogenetic excitation tool, an in vitro study.PloS one · 2024Article
- Nanoparticle-based optical interfaces for retinal neuromodulation: a review.Frontiers in cellular neuroscience · 2024Review
- Review
- Optogenetics Sheds Light on Brown and Beige Adipocytes.Journal of cellular signaling · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Optogenetic gene therapies offer a promising strategy for restoring vision to patients with retinal degenerative diseases, such as retinitis pigmentosa (RP). Several clinical trials have begun in this area using different vectors and optogenetic proteins (Clinical Identifiers: NCT02556736, NCT03326336, NCT04945772, and NCT04278131). Here we present preclinical efficacy and safety data for the NCT04278131 trial, which uses an AAV2 vector and Chronos as the optogenetic protein. Efficacy was assessed in mice in a dose-dependent manner using electroretinograms (ERGs). Safety was assessed in rats, nonhuman primates, and mice, using several tests, including immunohistochemical analyses and cell counts (rats), electroretinograms (nonhuman primates), and ocular toxicology assays (mice). The results showed that Chronos-expressing vectors were efficacious over a broad range of vector doses and stimulating light intensities, and were well tolerated: no test article-related findings were observed in the anatomical and electrophysiological assays performed.
Indexed as
Identifiers
What OpenQuestion holds
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.