ArticleThe Journal of clinical investigation2015
It's never too late to save a photoreceptor.
Article in The Journal of clinical investigation, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
7 citing papers in PubMed, 7 citations in OpenAlex.
- Proteomic analysis of CD29+ Müller cells reveals metabolic reprogramming in rabbit myopia model.Scientific reports · 2024Article
- Contribution of Müller Cells in the Diabetic Retinopathy Development: Focus on Oxidative Stress and Inflammation.Antioxidants (Basel, Switzerland) · 2022Review
- 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
- The CRB1 Complex: Following the Trail of Crumbs to a Feasible Gene Therapy Strategy.Frontiers in neuroscience · 2017Review
- Gene and cell-based therapies for inherited retinal disorders: An update.American journal of medical genetics. Part C, Seminars in medical genetics · 2016Review
- Induced Pluripotent Stem Cell Therapies for Degenerative Disease of the Outer Retina: Disease Modeling and Cell Replacement.Journal of ocular pharmacology and therapeutics : the official journal of the Association for Ocular Pharmacology and Therapeutics · 2016Review
Corrections and comments
- Comment on
Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
Recent gene therapy progress has raised the possibility that vision loss caused by inherited retinal degeneration can be slowed or prevented. Unfortunately, patients are not usually diagnosed until enough degeneration has occurred that the deterioration in vision is noticeable. Therefore, effective gene therapy must halt degeneration to stabilize and preserve any remaining vision. Gene therapy methods currently in human clinical trials rely on subretinal or intravitreal injections of adeno-associated virus to deliver the therapeutic gene. To date, long-term results in patients treated with subretinal injections for Leber congenital amaurosis have been mixed. Proposed limitations include variability in the gene delivery method and a possible point of no return, at which treatment would be ineffective. In this issue of the JCI, Koch et al. describe a well-controlled and precise mouse model for testing the ability of gene therapy to halt the progress of degeneration. Instead of viral-mediated therapeutic gene delivery, the authors induced expression of an integrated transgene at specific times during the course of photoreceptor degeneration. In Pde6b-deficient retina, this strategy halted degeneration, even when more than 70% of photoreceptors had already degenerated. The results of this study demonstrate that retinal degeneration can be stopped, even at late stages of disease.
Indexed as
Identifiers
What OpenQuestion holds
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.