ReviewThe application of clinical genetics2023
Adeno-Associated Virus (AAV) - Based Gene Therapies for Retinal Diseases: Where are We?
Review in The application of clinical genetics, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 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
26 citing papers in PubMed, 41 citations in OpenAlex.
- Restorative and regenerative therapies for vision loss.Eye (London, England) · 2026Review
- VEGFA-Targeted M3-F4 Ionizable Lipid Nanoparticles Improve Diabetic Retinopathy.Molecular pharmaceutics · 2026Article
- Precision Medicine in Inherited Retinal Disease: Advances, Challenges, and Future Directions.Journal of personalized medicine · 2026Review
- Bridging the gap: an emerging link between tubulinopathies and ciliopathies.NPJ genomic medicine · 2026Review
- Viral vector-based gene therapies in the clinic: An update.Bioengineering & translational medicine · 2026Review
- Combined AAV-mediated specific Gjb2 expression restores hearing in DFNB1 mouse models.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Toward improved AAV gene therapies for retinal disorders: challenges and advances.Regenerative medicine · 2025Review
- Face and Available Chair Detection and Localization With a Second-Generation (44-Channel) Suprachoroidal Retinal Prosthesis.Translational vision science & technology · 2025Observational
- The clinical safety landscape for ocular AAV gene therapies: A systematic review and meta-analysis.iScience · 2025Article
- mTOR downregulation promotes anti-inflammatory responses via the CCL3-CCR5 axis in hypoxic retinopathy.Molecular therapy. Methods & clinical development · 2025Article
- Emerging strategies targeting genes and cells in glaucoma.Vision research · 2025Review
- Article
- An Engineered Intravitreal Injection Retinal-Pigment-Epithelium-Tropic Adeno-Associated Virus Vector Expressing a Bispecific Antibody Binding VEGF-A and ANG-2 Rescues Neovascular Age-Related Macular Degeneration in Animal Models and Patients.Research (Washington, D.C.) · 2025Article
- Agarose hydrogel-mediated electroporation method for retinal tissue cultured at the air-liquid interface.iScience · 2024Article
- Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration.Molecular therapy. Methods & clinical development · 2024Article
- The Role of Artificial Intelligence and Machine Learning in Accelerating the Discovery and Development of Nanomedicine.Pharmaceutical research · 2024Article
- Characterization of anti-AAV2 neutralizing antibody levels in sheep prior to and following intravitreal AAV2.7m8 injection.Gene therapy · 2024Article
- Infantile Nystagmus Syndrome-Associated Inherited Retinal Diseases: Perspectives from Gene Therapy Clinical Trials.Life (Basel, Switzerland) · 2024Review
- Temporal insights into molecular and cellular responses during rAAV production in HEK293T cells.Molecular therapy. Methods & clinical development · 2024Article
- The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2.Gene therapy · 2024Article
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 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Owing to their small size and safety profiles, adeno-associated viruses (AAVs) have become the vector of choice for gene therapy applications in the retina. In addition to the naturally occurring AAVs, several engineered variants with enhanced properties are being developed for experimental and therapeutic applications. Nonetheless, there are still some challenges impeding successful application of AAVs for a broader range of retinal gene therapies. The small size of AAV particles ensures efficient tissue transduction but also limits the packaging capacity to a few kilobases. Further, AAV's ability to cross retinal barriers is still an obstacle to pan-retinal transduction of the outer retina with tolerable doses. Lastly, despite overall safety, there have been recent reports of immune responses to AAVs in the eye. Hence, evaluation and prediction of immune responses to AAVs has come to be considered an integral part of future clinical success. This review focuses on the use of AAV in clinical trials for retinal diseases, and discusses developments of variants and novel strategies to overcome immune responses to AAVs.
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.