ArticleMolecular therapy. Methods & clinical development2015
Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho(-/-) mouse.
Article in Molecular therapy. Methods & clinical development, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 12 papers.
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
12 citing papers in PubMed.
- Optimisation of AAV-NDI1 Significantly Enhances Its Therapeutic Value for Correcting Retinal Mitochondrial Dysfunction.Pharmaceutics · 2023Article
- AAV for Gene Therapy in Ocular Diseases: Progress and Prospects.Research (Washington, D.C.) · 2023Review
- AAV-PHP.eB transduces both the inner and outer retina with high efficacy in mice.Molecular therapy. Methods & clinical development · 2022Article
- Delivering AAV to the Central Nervous and Sensory Systems.Trends in pharmacological sciences · 2021Review
- Advanced late-onset retinitis pigmentosa with dominant-acting D477G RPE65 mutation is responsive to oral synthetic retinoid therapy.BMJ open ophthalmology · 2020Article
- AAV-DeliveredFrontiers in neuroscience · 2020Article
- Progress in Gene Therapy for Rhodopsin Autosomal Dominant Retinitis Pigmentosa.Advances in experimental medicine and biology · 2019Review
- Rod Outer Segment Development Influences AAV-Mediated Photoreceptor Transduction After Subretinal Injection.Human gene therapy · 2017Article
- A Novel Retinal Ganglion Cell Promoter for Utility in AAV Vectors.Frontiers in neuroscience · 2017Article
- Gene therapy approaches for the treatment of retinal disorders.Discovery medicine · 2016Review
- Advances in Gene Therapy for Diseases of the Eye.Human gene therapy · 2016Review
- Gene therapy to treat inherited and complex retinal degenerative diseases.Molecular therapy. Methods & clinical development · 2015Article
Corrections and comments
- Erratum issued
Authors and funding
10 authors.
Funding
Abstract
As gene therapies for various forms of retinal degeneration progress toward human clinical trial, it will be essential to have a repertoire of safe and efficient vectors for gene delivery to the target cells. Recombinant adeno-associated virus (AAV) serotype 2/2 has been shown to be well tolerated in the human retina and has provided efficacy in human patients for some inherited retinal degenerations. In this study, the AAV2/8 and AAV2/rh10 serotypes have been compared as a means of gene delivery to mammalian photoreceptor cells using a photoreceptor specific promoter for transgene expression. Both AAV2/8 and AAV2/rh10 provided rescue of the retinal degeneration present in the rhodopsin knockout mouse, with similar levels of benefit as evaluated by molecular, histological, and functional readouts. Transgene expression levels were significantly higher (fivefold) 1 week postsubretinal injection when employing AAV2/8 for rhodopsin gene delivery compared to AAV2/rh10, and were indistinguishable by 6 weeks postadministration of vector. This study reports the use of the AAV2/rh10 serotype to provide rescue in a degenerating retina and provides a comparative evaluation of AAV2/rh10 with respect to AAV2/8, a serotype regarded as providing efficient delivery to photoreceptors.
Identifiers
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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.