ArticleHuman molecular genetics2015
Vitreal delivery of AAV vectored Cnga3 restores cone function in CNGA3-/-/Nrl-/- mice, an all-cone model of CNGA3 achromatopsia.
Article in Human molecular genetics, 2015. 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
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Who cites it
19 citing papers in PubMed, 25 citations in OpenAlex.
- Gene therapy rescues cone function inMolecular therapy. Advances · 2026Article
- Gene therapy rescues cone function in an all-cone retina mouse model with the most common cone opsin C203R missense mutation.PloS one · 2026Article
- Nanomaterial-enabled delivery of plant-derived bioactive metabolites for diabetic retinopathy: from evidence appraisal to preclinical translation.Frontiers in pharmacology · 2026Review
- Biology, Pathobiology and Gene Therapy of CNG Channel-Related Retinopathies.Biomedicines · 2023Review
- Achromatopsia: Genetics and Gene Therapy.Molecular diagnosis & therapy · 2022Review
- Retinal Degeneration Caused by Ago2 Disruption.Investigative ophthalmology & visual science · 2021Article
- Gene therapy in color vision deficiency: a review.International ophthalmology · 2021Review
- AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration.Gene therapy · 2019Article
- AAV-mediated human CNGB3 restores cone function in an all-cone mouse model ofJournal of biomedical research · 2019Article
- Delivery of CR2-fH Using AAV Vector Therapy as Treatment Strategy in the Mouse Model of Choroidal Neovascularization.Molecular therapy. Methods & clinical development · 2018Article
- Retinal Cyclic Nucleotide-Gated Channels: From Pathophysiology to Therapy.International journal of molecular sciences · 2018Review
- Clinical and Genetic Evaluation of a Cohort of Pediatric Patients with Severe Inherited Retinal Dystrophies.Genes · 2017Article
- Gene-based Therapy in a Mouse Model of Blue Cone Monochromacy.Scientific reports · 2017Article
- Long-term retinal cone rescue using a capsid mutant AAV8 vector in a mouse model of CNGA3-achromatopsia.PloS one · 2017Article
- The Degeneration and Apoptosis Patterns of Cone Photoreceptors inJournal of ophthalmology · 2017Article
- Residual Foveal Cone Structure in CNGB3-Associated Achromatopsia.Investigative ophthalmology & visual science · 2016Article
- Animal modelling for inherited central vision loss.The Journal of pathology · 2016Review
- From bedside to bench--meeting report of the 7th International Conference on cGMP "cGMP: generators, effectors and therapeutic implications" in Trier, Germany, from June 19th to 21st 2015.Naunyn-Schmiedeberg's archives of pharmacology · 2015Review
- Canine CNGA3 Gene Mutations Provide Novel Insights into Human Achromatopsia-Associated Channelopathies and Treatment.PloS one · 2015Article
Corrections and comments
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Authors and funding
18 authors at 7 institutions in 3 countries.
Funding
Abstract
The CNGA3(-/-)/Nrl(-/-) mouse is a cone-dominant model with Cnga3 channel deficiency, which partially mimics the all cone foveal structure of human achromatopsia 2 with CNGA3 mutations. Although subretinal (SR) AAV vector administration can transfect retinal cells efficiently, the injection-induced retinal detachment can cause retinal damage, particularly when SR vector bleb includes the fovea. We therefore explored whether cone function-structure could be rescued in CNGA3(-/-)/Nrl(-/-) mice by intravitreal (IVit) delivery of tyrosine to phenylalanine (Y-F) capsid mutant AAV8. We find that AAV-mediated CNGA3 expression can restore cone function and rescue structure following IVit delivery of AAV8 (Y447, 733F) vector. Rescue was assessed by restoration of the cone-mediated electroretinogram (ERG), optomotor responses, and cone opsin immunohistochemistry. Demonstration of gene therapy in a cone-dominant mouse model by IVit delivery provides a potential alternative vector delivery mode for safely transducing foveal cones in achromatopsia patients and in other human retinal diseases affecting foveal function.
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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.