ReviewHuman gene therapy2015
Gene therapy of inherited retinal degenerations: prospects and challenges.
Review in Human gene therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 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
28 citing papers in PubMed, 47 citations in OpenAlex.
- Structure-Based Network Analysis of AlphaFold Structure Predictions Identifies Putative Causative Variants of Inherited Retinal Disease.Investigative ophthalmology & visual science · 2026Article
- Phagocytosis by retinal pigment epithelium and microglia does not affect vision restoration by P3HT nanoparticles in Retinitis pigmentosa.Cell death & disease · 2026Article
- Protection of GAP-43 gene-modified bone marrow mesenchymal stem cells on retinitis pigmentosa.Journal of translational medicine · 2026Article
- Laser photocoagulation improves the transduction efficiency of lentivirus vectors injected intravitreally.International journal of ophthalmology · 2026Article
- Graphene oxide increases the phototransduction efficiency of copolymeric nanoimplants and rescues visual functions in rat and pig models of Retinitis pigmentosa.Nature communications · 2025Article
- Optimized Minimally Invasive Transscleral Subretinal Injection Technique in Mouse.Journal of visualized experiments : JoVE · 2025Article
- A non-surgical method for subretinal delivery by trans-scleral microneedle injection.Bioengineering & translational medicine · 2025Article
- A membrane-targeted photoswitch restores physiological ON/OFF responses to light in the degenerate retina.Nature communications · 2025Article
- Nanomedicine and drug delivery to the retina: current status and implications for gene therapy.Naunyn-Schmiedeberg's archives of pharmacology · 2022Review
- Subretinal Injection Techniques for Retinal Disease: A Review.Journal of clinical medicine · 2022Review
- A New Mouse Model for Complete Congenital Stationary Night Blindness Due toInternational journal of molecular sciences · 2021Article
- Perspectives, Expectations, and Concerns of European Patient Advocates on Advanced Therapy Medicinal Products.Frontiers in medicine · 2021Article
- Microglia modulation by TGF-β1 protects cones in mouse models of retinal degeneration.The Journal of clinical investigation · 2020Article
- Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques.Molecular diagnosis & therapy · 2019Review
- Adeno-Associated Viral Gene Therapy for Inherited Retinal Disease.Pharmaceutical research · 2019Review
- The Eyes Have it: A Rheumatologist's View of Uveitis.Arthritis & rheumatology (Hoboken, N.J.) · 2018Review
- Review
- Phenotypic characterization of P23H and S334ter rhodopsin transgenic rat models of inherited retinal degeneration.Experimental eye research · 2018Article
- Recent Advancements in Gene Therapy for Hereditary Retinal Dystrophies.Turkish journal of ophthalmology · 2017Review
- Toward smart design of retinal drug carriers: a novel bovine retinal explant model to study the barrier role of the vitreoretinal interface.Drug delivery · 2017Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 1 institution in 1 country.
Funding
Abstract
Because of its favorable anatomical and immunological characteristics, the eye has been at the forefront of translational gene therapy. Dozens of promising proofs of concept have been obtained in animal models of inherited retinal degenerations (IRDs), and some of them have been relayed to the clinic. The results from the first clinical trials for a congenital form of blindness have generated great interest and have demonstrated the safety and efficacy of intraocular administrations of viral vectors in humans. However, this progress has also generated new questions and posed challenges that need to be addressed to further expand the applicability of gene therapy in the eye, including safe delivery of viral vectors to the outer retina, treatment of dominant IRDs as well as of IRDs caused by mutations in large genes, and, finally, selection of the appropriate IRDs and patients to maximize the efficacy of gene transfer. This review summarizes the strategies that are currently being exploited to overcome these challenges and drive the clinical development of retinal gene therapy.
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.