ArticleTranslational vision science & technology2020
Assessment of AAV Dual Vector Safety in the
Article in Translational vision science & technology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 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
12 citing papers in PubMed, 19 citations in OpenAlex.
- The Co-Transduction Conundrum: Improving Dual rAAV Delivery Through Co-Administration of Capsid Serotypes With Complementary Tropism.Translational vision science & technology · 2025Article
- Deep phenotyping of eyes shut homolog-associated retinopathy based on visual impairment patterns.Frontiers in ophthalmology · 2025Article
- Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants inMolecular therapy. Nucleic acids · 2024Article
- Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Review
- Proof-of-concept for multiple AON delivery by a single U7snRNA vector to restore splicing defects in ABCA4.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Adeno-Associated Virus (AAV) - Based Gene Therapies for Retinal Diseases: Where are We?The application of clinical genetics · 2023Review
- Minicircle Delivery to the Neural Retina as a Gene Therapy Approach.International journal of molecular sciences · 2022Review
- ABCA4 c.859-25A>G, a Frequent Palestinian Founder Mutation Affecting the Intron 7 Branchpoint, Is Associated With Early-Onset Stargardt Disease.Investigative ophthalmology & visual science · 2022Article
- Evaluation of Local Rod and Cone Function in Stargardt Disease.Investigative ophthalmology & visual science · 2022Article
- Gene-agnostic therapeutic approaches for inherited retinal degenerations.Frontiers in molecular neuroscience · 2022Review
- Therapy Approaches for Stargardt Disease.Biomolecules · 2021Review
- Gene Therapy to the Retina and the Cochlea.Frontiers in neuroscience · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 2 institutions in 1 country.
Funding
Abstract
Purpose: Adeno-associated viral (AAV) gene therapy treatment for Stargardt disease currently requires a dual vector approach owing to the size of the ATP-binding cassette transporter family member gene ( Methods: Results: Subretinal delivery of the dual vector system and its comprising parts induced no structural or functional changes relative to paired uninjected eyes beyond those observed in the sham control cohort. Histologic changes were limited to the superior retina where the injection was performed. Electroretinography analysis confirmed the dual vector system inferred no functional changes beyond those observed in the sham control cohort. Conclusions: An optimized overlapping dual vector system for the treatment of Stargardt disease shows no additional signs of toxicity beyond those observed from a sham injection. Translational Relevance: This presentation of safety of a dual vector system for the treatment of Stargardt disease encourages its future use in clinical trial.
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.