ReviewInvestigative ophthalmology & visual science2023
Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions.
Review in Investigative ophthalmology & visual science, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 78 papers.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
78 citing papers in PubMed.
- A novel adeno-associated viral vector derived from human spleen isolate AAV.hu.S17.Journal, genetic engineering & biotechnology · 2026Article
- Subretinal rAAV2-based VEGF-Trap gene therapy for neovascular age-related macular degeneration: Preclinical assessment and phase 1 trial results.Cell reports. Medicine · 2026Article
- Delivery devices and container closure systems for co-formulated and co-administered biologics.Antibody therapeutics · 2026Review
- Update on the Management of ABCA4 Retinopathy (Stargardt Disease).Ophthalmology and therapy · 2026Review
- Article
- Engineered tRNA reduces vision loss in a mouse model of Leber congenital amaurosis.Signal transduction and targeted therapy · 2026Article
- Halofantrine protects photoreceptors in multiple models of retinal degeneration.Research square · 2026Article
- Assessment of AAV/polyketal chimeric nanoparticles for ocular gene transduction using an animal model.Drug delivery and translational research · 2026Article
- PreclinicalbioRxiv : the preprint server for biology · 2026Article
- Targeting AKT via SC79 for Photoreceptor Preservation in Retinitis Pigmentosa Mouse Models.Biomedicines · 2026Article
- Long-Term Tolerability and Safety of AAV5-Id3 Gene Therapy to Eyes.Translational vision science & technology · 2026Article
- Innovations in mRNA-Based Nanoparticle for the Treatment of Ocular Disorders: A Comprehensive Review.Current pharmaceutical design · 2026Review
- Advances in hydrogel-mediated gene therapy in ophthalmology: future directions and therapeutic potential.Regenerative biomaterials · 2026Review
- Gene Therapy for Inherited Retinal Disease: Current Strategies, Personalized Medicine, and Future Implications-A Comprehensive Review.Journal of personalized medicine · 2025Review
- The economic impact of retinal diseases for which gene therapy is emerging: a systematic literature review.Health economics review · 2025Review
- Visual Neurorestoration: An Expert Review of Current Strategies for Restoring Vision in Humans.Brain sciences · 2025Review
- Surgical Approaches to Retinal Gene Therapy: 2025 Update.Bioengineering (Basel, Switzerland) · 2025Review
- Applications of Modern Cell Therapies: The Latest Data in Ophthalmology.Life (Basel, Switzerland) · 2025Review
- A Practical Guide to Genetic Eye Conditions for Paediatricians.Journal of paediatrics and child health · 2025Review
- Retinal transduction profiling of diverse AAV serotypes via intravitreal injection.Journal of virology · 2025Article
18 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Since long before the first approval of gene therapy for retinal disease, ocular gene therapy has captured the hopes of patients, clinicians, and scientists alike. Indeed, the retina provides a unique system for studying and treating ocular diseases, and it holds the distinction as the first tissue targeted by an approved gene therapy for inherited disorders in the United States. There are many methods for addressing genetic diseases in the eyes using a wide range of potential delivery systems and vectors. However, despite the immense progress over the last several decades, both old and new challenges remain, such as the long-term effects of treatments, immunogenicity, targeting, and manufacturing. This review provides a discussion of the history of ocular gene therapy, the various gene therapy approaches, methods to deliver a gene directly to ocular tissues (including both routes of administration and vectors), challenges to ocular gene therapy, the current clinical trial landscape, and future directions of the field.
Indexed as
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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.