ArticleHuman gene therapy2010
Changes in adeno-associated virus-mediated gene delivery in retinal degeneration.
Article in Human gene therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 42 papers, 1 of them a synthesis that pooled it.
What it found
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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
42 citing papers in PubMed, 1 synthesis or guideline pooled it.
- 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
- Novel photoreceptor-specific promoters for gene therapy in mid- to late-stage retinal degeneration.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Intravitreal AAV2 gene delivery to feline retinal ganglion cells.Vision research · 2025Article
- Developing New Vectors for Retinal Gene Therapy.Cold Spring Harbor perspectives in medicine · 2023Article
- Intraocular neutralizing antibodies against aflibercept in patients with age related macular degeneration.BMC ophthalmology · 2023Article
- Gene therapy for inherited retinal diseases: exploiting new tools in genome editing and nanotechnology.Frontiers in ophthalmology · 2023Review
- Harnessing the Neuroprotective Behaviors of Müller Glia for Retinal Repair.Frontiers in bioscience (Landmark edition) · 2022Review
- Defining Phenotype, Tropism, and Retinal Gene Therapy Using Adeno-Associated Viral Vectors (AAVs) in New-Born Brown Norway Rats with a Spontaneous Mutation inInternational journal of molecular sciences · 2021Article
- Trans-Ocular Electric Current In Vivo Enhances AAV-Mediated Retinal Transduction in Large Animal Eye After Intravitreal Vector Administration.Translational vision science & technology · 2020Article
- Optogenetic Gene Therapy for the Degenerate Retina: Recent Advances.Frontiers in neuroscience · 2020Review
- AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration.Gene therapy · 2019Article
- Trans-ocular Electric CurrentMolecular therapy. Methods & clinical development · 2019Article
- Translational Retinal Research and Therapies.Translational vision science & technology · 2018Article
- Phenotypic characterization of P23H and S334ter rhodopsin transgenic rat models of inherited retinal degeneration.Experimental eye research · 2018Article
- Noninvasive gene delivery to foveal cones for vision restoration.JCI insight · 2018Article
- AAV2-Mediated Transduction of the Mouse Retina After Optic Nerve Injury.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
- Heparan Sulfate Binding Promotes Accumulation of Intravitreally Delivered Adeno-associated Viral Vectors at the Retina for Enhanced Transduction but Weakly Influences Tropism.Journal of virology · 2016Article
- Diabetes enhances the efficacy of AAV2 vectors in the retina: therapeutic effect of AAV2 encoding vasoinhibin and soluble VEGF receptor 1.Laboratory investigation; a journal of technical methods and pathology · 2016Article
- Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs.Gene therapy · 2016Article
Corrections and comments
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Authors and funding
7 authors.
Funding
Abstract
Gene therapies for retinal degeneration have relied on subretinal delivery of viral vectors carrying therapeutic DNA. The subretinal injection is clearly not ideal as it limits the viral transduction profile to a focal region at the injection site and negatively affects the neural retina by detaching it from the supportive retinal pigment epithelium (RPE). We assessed changes in adeno-associated virus (AAV) dispersion and transduction in the degenerating rat retina after intravitreal delivery. We observed a significant increase in AAV-mediated gene transfer in the diseased compared with normal retina, the extent of which depends on the AAV serotype injected. We also identified key structural changes that correspond to increased viral infectivity. Particle diffusion and transgene accumulation in normal and diseased retina were monitored via fluorescent labeling of viral capsids and quantitative PCR. Viral particles were observed to accumulate at the vitreoretinal junction in normal retina, whereas particles spread into the outer retina and RPE in degenerated tissue. Immunohistochemistry illustrates remarkable changes in the architecture of the inner limiting membrane, which are likely to underlie the increased viral transduction in diseased retina. These data highlight the importance of characterizing gene delivery vectors in diseased tissue as structural and biochemical changes can alter viral vector transduction patterns. Furthermore, these results indicate that gene delivery to the outer nuclear layer may be achieved by noninvasive intravitreal AAV administration in the diseased state.
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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.