ArticleProceedings of the National Academy of Sciences of the United States of America2017
Genetic rescue models refute nonautonomous rod cell death in retinitis pigmentosa.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2017. 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 22 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.
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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.
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Who cites it
22 citing papers in PubMed, 31 citations in OpenAlex.
- Nutritional supplements: current evidence for retinitis pigmentosa and Stargardt disease.Current opinion in ophthalmology · 2026Review
- Article
- Gene augmentation therapy restores vision and preserves photoreceptors in a mouse model of CNGA1-retinitis pigmentosa.Communications medicine · 2025Article
- Ablating VHL in rod photoreceptors modulates RPE glycolysis and improves preclinical model of retinitis pigmentosa.The Journal of clinical investigation · 2025Article
- CRISPR editing of anti-anemia drug target rescues independent preclinical models of retinitis pigmentosa.Cell reports. Medicine · 2024Article
- Late gene therapy limits the restoration of retinal function in a mouse model of retinitis pigmentosa.Nature communications · 2023Article
- A New Preclinical Model of Retinitis Pigmentosa Due toOphthalmology science · 2023Article
- Late gene therapy limits the restoration of retinal function in a mouse model of retinitis pigmentosa.bioRxiv : the preprint server for biology · 2023Article
- Rod Photoreceptor-Specific Ablation of Metformin Target, AMPK, in a Preclinical Model of Autosomal Recessive Retinitis Pigmentosa.Advances in experimental medicine and biology · 2023Article
- Long-term vitamin A supplementation in a preclinical mouse model for RhoD190N-associated retinitis pigmentosa.Human molecular genetics · 2022Article
- Depletion of miR-96 Delays, But Does Not Arrest, Photoreceptor Development in Mice.Investigative ophthalmology & visual science · 2022Article
- Late-stage rescue of visually guided behavior in the context of a significantly remodeled retinitis pigmentosa mouse model.Cellular and molecular life sciences : CMLS · 2022Article
- Renormalization of metabolic coupling treats age-related degenerative disorders: an oxidative RPE niche fuels the more glycolytic photoreceptors.Eye (London, England) · 2022Review
- Retina Metabolism and Metabolism in the Pigmented Epithelium: A Busy Intersection.Annual review of vision science · 2021Review
- Limited time window for retinal gene therapy in a preclinical model of ciliopathy.Human molecular genetics · 2020Article
- Rod function deficit in retained photoreceptors of patients with class B Rhodopsin mutations.Scientific reports · 2020Article
- Perspectives on Gene Therapy: Choroideremia Represents a Challenging Model for the Treatment of Other Inherited Retinal Degenerations.Translational vision science & technology · 2020Article
- Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses.Nature biomedical engineering · 2020Article
- CAPN5 genetic inactivation phenotype supports therapeutic inhibition trials.Human mutation · 2019Article
- Genetic Rescue Reverses Microglial Activation in Preclinical Models of Retinitis Pigmentosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
Corrections and comments
- Erratum issued
Authors and funding
7 authors at 5 institutions in 3 countries.
Funding
Abstract
Retinitis pigmentosa (RP) is an inherited neurodegenerative disease, in which the death of mutant rod photoreceptors leads secondarily to the non-cell autonomous death of cone photoreceptors. Gene therapy is a promising treatment strategy. Unfortunately, current methods of gene delivery treat only a fraction of diseased cells, yielding retinas that are a mosaic of treated and untreated rods, as well as cones. In this study, we created two RP mouse models to test whether dying, untreated rods negatively impact treated, rescued rods. In one model, treated and untreated rods were segregated. In the second model, treated and untreated rods were diffusely intermixed, and their ratio was controlled to achieve low-, medium-, or high-efficiency rescue. Analysis of these mosaic retinas demonstrated that rescued rods (and cones) survive, even when they are greatly outnumbered by dying photoreceptors. On the other hand, the rescued photoreceptors did exhibit long-term defects in their outer segments (OSs), which were less severe when more photoreceptors were treated. In summary, our study suggests that even low-efficiency gene therapy may achieve stable survival of rescued photoreceptors in RP patients, albeit with OS dysgenesis.
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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.