ArticleThe Journal of neuroscience : the official journal of the Society for Neuroscience2013
Therapeutic margins in a novel preclinical model of retinitis pigmentosa.
Article in The Journal of neuroscience : the official journal of the Society for Neuroscience, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. 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.
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.
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Who cites it
22 citing papers in PubMed, 38 citations in OpenAlex.
- Safety and vision outcomes of subretinal gene supplementation therapy inThe British journal of ophthalmology · 2026Trial
- Article
- Ablating VHL in rod photoreceptors modulates RPE glycolysis and improves preclinical model of retinitis pigmentosa.The Journal of clinical investigation · 2025Article
- CD44 signaling in Müller cells impacts photoreceptor function and survival in healthy and diseased retinas.Journal of neuroinflammation · 2024Article
- CRISPR editing of anti-anemia drug target rescues independent preclinical models of retinitis pigmentosa.Cell reports. Medicine · 2024Article
- The temporal progression of retinal degeneration and early-stage idebenone treatment in the Pde6bScientific reports · 2024Article
- Rod Photoreceptor-Specific Ablation of Metformin Target, AMPK, in a Preclinical Model of Autosomal Recessive Retinitis Pigmentosa.Advances in experimental medicine and biology · 2023Article
- 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
- Comprehensive structure-function analysis of causative variants in retinal pigment epithelium specific 65 kDa protein associated Leber Congenital Amaurosis.Non-coding RNA research · 2019Article
- Genetic Rescue Reverses Microglial Activation in Preclinical Models of Retinitis Pigmentosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Cochlear Gene Therapy for Sensorineural Hearing Loss: Current Status and Major Remaining Hurdles for Translational Success.Frontiers in molecular neuroscience · 2018Review
- Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy.Neural plasticity · 2018Review
- Genetic rescue models refute nonautonomous rod cell death in retinitis pigmentosa.Proceedings of the National Academy of Sciences of the United States of America · 2017Article
- Gene and cell-based therapies for inherited retinal disorders: An update.American journal of medical genetics. Part C, Seminars in medical genetics · 2016Review
- Reprogramming metabolism by targeting sirtuin 6 attenuates retinal degeneration.The Journal of clinical investigation · 2016Article
- CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- Green tea extract attenuates MNU-induced photoreceptor cell apoptosis via suppression of heme oxygenase-1.Journal of toxicologic pathology · 2016Article
- Susceptibility to N-methyl-N-nitrosourea-induced retinal degeneration in different rat strains.Journal of toxicologic pathology · 2016Article
- Halting progressive neurodegeneration in advanced retinitis pigmentosa.The Journal of clinical investigation · 2015Article
- Emerging Treatments for Retinitis Pigmentosa: Genes and stem cells, as well as new electronic and medical therapies, are gaining ground.Retinal physician · 2015Article
Corrections and comments
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Authors and funding
7 authors at 4 institutions in 4 countries.
Funding
Abstract
The third-most common cause of autosomal recessive retinitis pigmentosa (RP) is due to defective cGMP phosphodiesterase-6 (PDE6). Previous work using viral gene therapy on PDE6-mutant mouse models demonstrated photoreceptors can be rescued if administered before degeneration. However, whether visual function can be rescued after degeneration onset has not been addressed. This is a clinically important question, as newly diagnosed patients exhibit considerable loss of rods and cones in their peripheral retinas. We have generated and characterized a tamoxifen inducible Cre-loxP rescue allele, Pde6b(Stop), which allows us to temporally correct PDE6-deficiency. Whereas untreated mutants exhibit degeneration, activation of Cre-loxP recombination in early embryogenesis produced stable long-term rescue. Reversal at later time-points showed partial long-term or short-lived rescue. Our results suggest stable restoration of retinal function by gene therapy can be achieved if a sufficient number of rods are treated. Because patients are generally diagnosed after extensive loss of rods, the success of clinical trials may depend on identifying patients as early as possible to maximize the number of treatable rods.
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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.