ArticleGene therapy2016
Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4
Article in Gene therapy, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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
19 citing papers in PubMed, 30 citations in OpenAlex.
- [Remodeling of the internal retina-Implications for targeted optogenetics].Die Ophthalmologie · 2026Review
- Review
- Age-related macular degeneration: suitability of optogenetic therapy for geographic atrophy.Frontiers in neuroscience · 2024Review
- Emerging optogenetics technologies in biomedical applications.Smart medicine · 2023Review
- Optogenetic approaches to therapy for inherited retinal degenerations.The Journal of physiology · 2022Review
- Quantitative single-cell transcriptome-based ranking of engineered AAVs in human retinal explants.Molecular therapy. Methods & clinical development · 2022Article
- Tropism of AAV Vectors in Photoreceptor-Like Cells of Human iPSC-Derived Retinal Organoids.Translational vision science & technology · 2022Article
- The Direct Healthcare Cost of Stargardt Disease: A Claims-Based Analysis.Ophthalmic epidemiology · 2021Article
- The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease.International journal of molecular sciences · 2021Review
- A 2020 vision of ocular gene therapy.Gene therapy · 2021Article
- Repair of Retinal Degeneration following Ex Vivo Minicircle DNA Gene Therapy and Transplantation of Corrected Photoreceptor Progenitors.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- Optogenetic Gene Therapy for the Degenerate Retina: Recent Advances.Frontiers in neuroscience · 2020Review
- An AAV Dual Vector Strategy Ameliorates the Stargardt Phenotype in AdultHuman gene therapy · 2019Article
- Transduction Patterns of Adeno-associated Viral Vectors in a Laser-Induced Choroidal Neovascularization Mouse Model.Molecular therapy. Methods & clinical development · 2018Article
- Improvement of Photoreceptor Targeting via Intravitreal Delivery in Mouse and Human Retina Using Combinatory rAAV2 Capsid Mutant Vectors.Investigative ophthalmology & visual science · 2017Article
- Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina.Gene therapy · 2017Article
- Emerging Gene Therapies for Genetic Hearing Loss.Journal of the Association for Research in Otolaryngology : JARO · 2017Review
- Exosome-associated AAV2 vector mediates robust gene delivery into the murine retina upon intravitreal injection.Scientific reports · 2017Article
- Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human Retina.Molecular therapy. Nucleic acids · 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
9 authors at 3 institutions in 2 countries.
Funding
Abstract
Gene therapy using adeno-associated viral (AAV) vectors for the treatment of retinal degenerations has shown safety and efficacy in clinical trials. However, very high levels of vector expression may be necessary for the treatment of conditions such as Stargardt disease where a dual vector approach is potentially needed, or in optogenetic strategies for end-stage degeneration in order to achieve maximal light sensitivity. In this study, we assessed two vectors with single capsid mutations, rAAV2/2(Y444F) and rAAV2/8(Y733F) in their ability to transduce retina in the Abca4
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.