ReviewGenes2018
Revolution in Gene Medicine Therapy and Genome Surgery.
Review in Genes, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 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
15 citing papers in PubMed.
- RHO1-2 meganuclease gene editing targets human P23H rhodopsin-induced retinitis pigmentosa to rejuvenate rods and maintain cones.bioRxiv : the preprint server for biology · 2025Article
- Adeno-associated virus-clustered regularly interspaced short palindromic repeats/cas9‑mediated ovarian cancer treatment targeting PD-L1.BMC cancer · 2025Article
- Review
- Syndecan-4 Mediates the Cellular Entry of Adeno-Associated Virus 9.International journal of molecular sciences · 2023Article
- Development of clustered regularly interspaced short palindromic repeats/CRISPR-associated technology for potential clinical applications.World journal of clinical cases · 2022Review
- Harnessing the Neuroprotective Behaviors of Müller Glia for Retinal Repair.Frontiers in bioscience (Landmark edition) · 2022Review
- CRISPR/Cas therapeutic strategies for autosomal dominant disorders.The Journal of clinical investigation · 2022Review
- Acute spinal cord injury: Pathophysiology and pharmacological intervention (Review).Molecular medicine reports · 2021Review
- [Rapid screening of single guide RNA targeting pig genome and the harvesting of monoclonal cells by microarray seal].Sheng wu yi xue gong cheng xue za zhi = Journal of biomedical engineering = Shengwu yixue gongchengxue zazhi · 2021Article
- Microglia Activation and Inflammation During the Death of Mammalian Photoreceptors.Annual review of vision science · 2020Review
- Differences in Intraretinal Pigment Migration Across Inherited Retinal Dystrophies.American journal of ophthalmology · 2020Article
- Sensitive Oligodeoxynucleotide Synthesis Using Dim and Dmoc as Protecting Groups.The Journal of organic chemistry · 2019Article
- Medicinal Chemistry: From Targets to Therapies.ACS medicinal chemistry letters · 2019Article
- Modeling Host-Virus Interactions in Viral Infectious Diseases Using Stem-Cell-Derived Systems and CRISPR/Cas9 Technology.Viruses · 2019Review
- Systematic Profiling of Alternative mRNA Splicing Signature for Predicting Glioblastoma Prognosis.Frontiers in oncology · 2019Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
Recently, there have been revolutions in the development of both gene medicine therapy and genome surgical treatments for inherited disorders. Much of this progress has been centered on hereditary retinal dystrophies, because the eye is an immune-privileged and anatomically ideal target. Gene therapy treatments, already demonstrated to be safe and efficacious in numerous clinical trials, are benefitting from the development of new viral vectors, such as dual and triple adeno-associated virus (AAV) vectors. CRISPR/Cas9, which revolutionized the field of gene editing, is being adapted into more precise "high fidelity" and catalytically dead variants. Newer CRISPR endonucleases, such as CjCas9 and Cas12a, are generating excitement in the field as well. Stem cell therapy has emerged as a promising alternative, allowing human embryo-derived stem cells and induced pluripotent stem cells to be edited precisely in vitro and then reintroduced into the body. This article highlights recent progress made in gene therapy and genome surgery for retinal disorders, and it provides an update on precision medicine Food and Drug Administration (FDA) treatment trials.
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.