ArticleInvestigative ophthalmology & visual science2010
Engineered zinc finger nuclease-mediated homologous recombination of the human rhodopsin gene.
Article in Investigative ophthalmology & visual science, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 24 citations in OpenAlex.
- Non-Viral Delivery Systems to Transport Nucleic Acids for Inherited Retinal Disorders.Pharmaceuticals (Basel, Switzerland) · 2025Review
- Gene Therapy for Retinitis Pigmentosa: Current Challenges and New Progress.Biomolecules · 2024Review
- Genome editing, a superior therapy for inherited retinal diseases.Vision research · 2023Article
- Harnessing the Neuroprotective Behaviors of Müller Glia for Retinal Repair.Frontiers in bioscience (Landmark edition) · 2022Review
- Gene regulatory and gene editing tools and their applications for retinal diseases and neuroprotection: From proof-of-concept to clinical trial.Frontiers in neuroscience · 2022Review
- Molecular Therapies for Inherited Retinal Diseases-Current Standing, Opportunities and Challenges.Genes · 2019Review
- Genome Editing as a Treatment for the Most Prevalent Causative Genes of Autosomal Dominant Retinitis Pigmentosa.International journal of molecular sciences · 2019Review
- Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy.Neural plasticity · 2018Review
- Gene and cell-based therapies for inherited retinal disorders: An update.American journal of medical genetics. Part C, Seminars in medical genetics · 2016Review
- Genome Engineering with TALE and CRISPR Systems in Neuroscience.Frontiers in genetics · 2016Review
- Non-viral therapeutic approaches to ocular diseases: An overview and future directions.Journal of controlled release : official journal of the Controlled Release Society · 2015Review
- Gene therapy in animal models of autosomal dominant retinitis pigmentosa.Molecular vision · 2012Review
Corrections and comments
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
purposeNovel zinc finger nucleases (ZFNs) were designed to target the human rhodopsin gene and induce homologous recombination of a donor DNA fragment.
methodsThree-finger zinc finger nucleases were designed based on previously published guidelines. To assay for ZFN specificity, the authors generated human embryonic retinoblast cell lines stably expressing a Pro23His rhodopsin, the most common mutation associated with autosomal dominant retinitis pigmentosa in North America. They report quantification of these rhodopsin-specific ZFNs to induce a targeted double-strand break in the human genome, demonstrate their ability to induce homologous recombination of a donor DNA fragment, and report the quantification of the frequency of ZFN-mediated homologous recombination.
resultsCompared with endogenous homologous recombination, the authors observed a 12-fold increase in homologous recombination and an absolute frequency of ZFN-directed homologous recombination as high as 17% in the human rhodopsin gene.
conclusionsZFNs are chimeric proteins with significant potential for the treatment of inherited diseases. In this study, the authors report the design of novel ZFNs targeting the human rhodopsin gene. These ZFNs may be useful for the treatment of retinal diseases such as retinitis pigmentosa, one of the most common causes of inherited blindness in the developed world. Herein, they also report on several aspects of donor fragment design and in vitro conditions that facilitate ZFN-mediated homologous recombination.
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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.