ReviewTranslational research : the journal of laboratory and clinical medicine2013
Gene therapy for retinal disease.
Review in Translational research : the journal of laboratory and clinical medicine, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers, 1 of them a synthesis that pooled it.
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
32 citing papers in PubMed, 1 synthesis or guideline pooled it, 73 citations in OpenAlex.
- Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays.International journal of molecular sciences · 2020Pooled it
- Long-Term Results from an Epiretinal Prosthesis to Restore Sight to the Blind.Ophthalmology · 2015Trial
- Modalities of vision restoration in optic neuropathies and retinal disease.Progress in retinal and eye research · 2026Review
- Prominin-1 and Retinal Degenerative Disorders: Expanding the Biology from Photoreceptors to the Retinal Pigment Epithelium.Biomolecules · 2026Review
- Progress and prospect of minicircle as a minimized non-viral DNA vector in gene therapy and regenerative medicine.Molecular therapy. Nucleic acids · 2025Review
- Gene Therapies in Clinical Development to Treat Retinal Disorders.Molecular diagnosis & therapy · 2024Review
- Intravitreal Delivery of PEGylated-ECO Plasmid DNA Nanoparticles for Gene Therapy of Stargardt Disease.Pharmaceutical research · 2024Article
- Long-Term Porcine Retina Explants as an Alternative to In Vivo Experimentation.Translational vision science & technology · 2024Article
- The Role of Subretinal Injection in Ophthalmic Surgery: Therapeutic Agent Delivery and Other Indications.International journal of molecular sciences · 2023Review
- Nanoparticles-mediated CRISPR-Cas9 gene therapy in inherited retinal diseases: applications, challenges, and emerging opportunities.Journal of nanobiotechnology · 2022Review
- Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders.EMBO molecular medicine · 2021Article
- An analysis of the Kozak consensus in retinal genes and its relevance to gene therapy.Molecular vision · 2021Article
- Differences in Intraretinal Pigment Migration Across Inherited Retinal Dystrophies.American journal of ophthalmology · 2020Article
- Niosomal Drug Delivery Systems for Ocular Disease-Recent Advances and Future Prospects.Nanomaterials (Basel, Switzerland) · 2020Review
- CHOROIDEREMIA: Retinal Degeneration With an Unmet Need.Retina (Philadelphia, Pa.) · 2019Review
- Molecular Therapies for Inherited Retinal Diseases-Current Standing, Opportunities and Challenges.Genes · 2019Review
- Assessment of Postoperative Morphologic Retinal Changes by Optical Coherence Tomography in Recipients of an Electronic Retinal Prosthesis Implant.JAMA ophthalmology · 2019Article
- Adeno-Associated Viral Gene Therapy for Inherited Retinal Disease.Pharmaceutical research · 2019Review
- The Application of CRISPR/Cas9 for the Treatment of Retinal Diseases.The Yale journal of biology and medicine · 2017Review
- Development of the Ultra-Low Vision Visual Functioning Questionnaire (ULV-VFQ).Translational vision science & technology · 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
2 authors at 2 institutions in 1 country.
Funding
Abstract
Gene therapy strategies for the treatment of inherited retinal diseases have made major advances in recent years. This review focuses on adeno-associated viral (AAV) vector approaches to treat retinal degeneration and, thus, prevent or delay the onset of blindness. Data from human clinical trials of gene therapy for retinal disease show encouraging signs of safety and efficacy from AAV vectors. Recent progress in enhancing cell-specific targeting and transduction efficiency of the various retinal layers plus the use of AAV-delivered growth factors to augment the therapeutic effect and limit cell death suggest even greater success in future human trials is possible.
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.