ArticleMolecular therapy : the journal of the American Society of Gene Therapy2010
Zinc-finger nuclease-induced gene repair with oligodeoxynucleotides: wanted and unwanted target locus modifications.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 55 papers.
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Who cites it
55 citing papers in PubMed, 128 citations in OpenAlex.
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- CRISPR-Cas9 editing of a TNPO3 mutation in a muscle cell model of limb-girdle muscular dystrophy type D2.Molecular therapy. Nucleic acids · 2023Article
- CRISPR-Cas9‒Based Genomic Engineering in Keratinocytes: From Technology to Application.JID innovations : skin science from molecules to population health · 2022Review
- Modified Gene Editing Systems: Diverse Bioengineering Tools and Crop Improvement.Frontiers in plant science · 2022Review
- Extensive trimming of short single-stranded DNA oligonucleotides during replication-coupled gene editing in mammalian cells.PLoS genetics · 2020Article
- Mitigating off-target effects in CRISPR/Cas9-mediated in vivo gene editing.Journal of molecular medicine (Berlin, Germany) · 2020Review
- Template plasmid integration in germline genome-edited cattle.Nature biotechnology · 2020Article
- Article
- High-frequency random DNA insertions upon co-delivery of CRISPR-Cas9 ribonucleoprotein and selectable marker plasmid in rice.Scientific reports · 2019Article
- High Efficiency Targeting of Non-coding Sequences Using CRISPR/Cas9 System in Tilapia.G3 (Bethesda, Md.) · 2019Article
- Delivering CRISPR: a review of the challenges and approaches.Drug delivery · 2018Review
- Generation of genetically-engineered animals using engineered endonucleases.Archives of pharmacal research · 2018Review
- DNA mismatch repair and oligonucleotide end-protection promote base-pair substitution distal from a CRISPR/Cas9-induced DNA break.Nucleic acids research · 2018Article
- Repair of a Site-Specific DNA Cleavage: Old-School Lessons for Cas9-Mediated Gene Editing.ACS chemical biology · 2018Review
- Genome-wide Mapping of Off-Target Events in Single-Stranded Oligodeoxynucleotide-Mediated Gene Repair Experiments.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Relaxed chromatin induced by histone deacetylase inhibitors improves the oligonucleotide-directed gene editing in plant cells.Journal of plant research · 2018Article
- USH2A Gene Editing Using the CRISPR System.Molecular therapy. Nucleic acids · 2017Article
- A history of genome editing in mammals.Mammalian genome : official journal of the International Mammalian Genome Society · 2017Review
- Genome editing revolutionize the creation of genetically modified pigs for modeling human diseases.Human genetics · 2016Review
- Emerging therapies for mitochondrial disorders.Brain : a journal of neurology · 2016Review
Corrections and comments
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Authors and funding
4 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Correcting a mutated gene directly at its endogenous locus represents an alternative to gene therapy protocols based on viral vectors with their risk of insertional mutagenesis. When solely a single-stranded oligodeoxynucleotide (ssODN) is used as a repair matrix, the efficiency of the targeted gene correction is low. However, as shown with the homing endonuclease I-SceI, ssODN-mediated gene correction can be enhanced by concomitantly inducing a DNA double-strand break (DSB) close to the mutation. Because I-SceI is hardly adjustable to cut at any desired position in the human genome, here, customizable zinc-finger nucleases (ZFNs) were used to stimulate ssODN-mediated repair of a mutated single-copy reporter locus stably integrated into human embryonic kidney-293 cells. The ZFNs induced faithful gene repair at a frequency of 0.16%. Six times more often, ZFN-induced DSBs were found to be modified by unfaithful addition of ssODN between the termini and about 60 times more often by nonhomologous end joining-related deletions and insertions. Additionally, ZFN off-target activity based on binding mismatch sites at the locus of interest was detected in in vitro cleavage assays and also in chromosomal DNA isolated from treated cells. Therefore, the specificity of ZFN-induced ssODN-mediated gene repair needs to be improved, especially regarding clinical applications.
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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.