ReviewEuropean journal of pediatrics2012
Retinal dystrophies and gene therapy.
Review in European journal of pediatrics, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 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
17 citing papers in PubMed, 24 citations in OpenAlex.
- Parents' perceptions of diagnostic genetic testing for children with inherited retinal disease in China.Molecular genetics & genomic medicine · 2019Trial
- Gene Therapy in Hereditary Retinal Dystrophies: The Usefulness of Diagnostic Tools in Candidate Patient Selections.International journal of molecular sciences · 2023Review
- Non-Viral Delivery of CRISPR/Cas Cargo to the Retina Using Nanoparticles: Current Possibilities, Challenges, and Limitations.Pharmaceutics · 2022Review
- Newer therapeutic options for inherited retinal diseases: Gene and cell replacement therapy.Indian journal of ophthalmology · 2022Review
- NGS and phenotypic ontology-based approaches increase the diagnostic yield in syndromic retinal diseases.Human genetics · 2021Article
- An Overview of the Genetics ofGenes · 2021Review
- Genotype-Phenotype Analysis ofFrontiers in genetics · 2021Article
- Morpho-functional survey in children suspected of inherited retinal dystrophies via video recording, electrophysiology and genetic analysis.International ophthalmology · 2020Article
- CRISPR GENOME SURGERY IN THE RETINA IN LIGHT OF OFF-TARGETING.Retina (Philadelphia, Pa.) · 2018Review
- Laboratory and clinical reliability of conformally coated subretinal implants.Biomedical microdevices · 2017Article
- New and emerging technologies for the treatment of inherited retinal diseases: a horizon scanning review.Eye (London, England) · 2015Review
- Lipid Nanoparticles for Ocular Gene Delivery.Journal of functional biomaterials · 2015Review
- Review and update on the molecular basis of Leber congenital amaurosis.World journal of clinical cases · 2015Review
- The 2014 Bowman Lecture-Bowman's and Bruch's: a tale of two membranes during the laser revolution.Eye (London, England) · 2015Article
- Combined rod and cone transduction by adeno-associated virus 2/8.Human gene therapy · 2013Article
- The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.Human gene therapy · 2012Article
- Translational Epidemiology in Ophthalmology: From Etiologic Research to Personalized Health Impact.Translational vision science & technology · 2012Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 2 institutions in 1 country.
Funding
Abstract
Retinal dystrophies are inherited disorders of photoreceptor and retinal pigment epithelial function that may result in severe visual impairment. Advances in molecular genetics have helped identify many of the gene defects responsible, and progress in gene transfer technology has enabled therapeutic strategies to be developed and applied. The first human clinical trials of gene therapy for RPE65 associated retinal dystrophy have shown promising initial results and have helped prepare the way for further trials of gene therapy for inherited retinal disorders. The results of these trials will provide further insight into the safety and efficacy of gene therapy for a range of currently untreatable and debilitating eye disorders.
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.