ArticleNature biotechnology2013
High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells.
Article in Nature biotechnology, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1,837 papers, 1 of them a synthesis that pooled it.
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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.
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Who cites it
1,837 citing papers in PubMed, 1 synthesis or guideline pooled it.
- The evolving landscape of gene editing therapies for human genetic diseases: a twenty-year bibliometric analysis.Frontiers in medicine · 2026Pooled it
- CAMKV kinase drives neuroblastoma growth and acts as a novel therapeutic target.The Journal of experimental medicine · 2026Article
- CRISPR in clinical oncology: translational advances from molecular diagnostics to therapeutics.Nature reviews. Clinical oncology · 2026Review
- A dynamically frustrated allosteric checkpoint consistent with conformational proofreading in CRISPR-Cas9.Nucleic acids research · 2026Article
- Size and sequence variation of the Lid of the Cas12a nuclease domain results in a nickase phenotype.Nucleic acids research · 2026Article
- Accurate characterization of CRISPR-Cas9 genome editing outcomes and mosaicism with near-perfect long reads.Genome medicine · 2026Article
- Article
- Site-specific gene integration by recombinase-mediated cassette exchange in anhydrobiotic Pv11 cells.In vitro cellular & developmental biology. Animal · 2026Article
- Miniaturized CRISPR: Ultra Compact Systems for In Vivo Delivery and Portable Diagnostics.Annals of biomedical engineering · 2026Review
- Engineering editopes through programmable RNA editing toward tumor neoantigen generation.The EMBO journal · 2026Article
- Single-cell and in vivo profiling reveal heterogeneous and organ-specific CRISPR-Cas9 off-target and translocation outcomes.Nature communications · 2026Article
- Analysing long-read CRISPR experiments with CRISPRLungo.Nature biomedical engineering · 2026Article
- Compact 9dBEs Enable Efficient and Precise Genome Editing in Mammalian Cells and In Vivo.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Applications of synthetic biology in biomedicine.Molecular biomedicine · 2026Review
- Precision Lipid Management in the Era of Biotechnology and Artificial Intelligence: From Gene Editing to Smart Drug Delivery.Therapeutic innovation & regulatory science · 2026Review
- Improvement of precise large-fragment deletion with a versatile nuclease prime editor.Cell discovery · 2026Article
- UNCOVERseq enables sensitive and controlled gene editing off-target nomination across CRISPR-Cas modalities and systems.Nature communications · 2026Article
- Specific cleavage of 5' overhangs of non-target strand by Cas9 activated by target DNA binding.Nucleic acids research · 2026Article
- Upper bound on the mutational burden imposed by a CRISPR-Cas9 gene-drive element.G3 (Bethesda, Md.) · 2026Article
- abCRISPR: deep learning-based design of abasic gRNA sequences for specific CRISPR-Cas9 genome editing.Bioinformatics (Oxford, England) · 2026Article
1,777 more citing papers are in PubMed but not listed here.
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7 authors.
Funding
Abstract
Clustered, regularly interspaced, short palindromic repeat (CRISPR) RNA-guided nucleases (RGNs) have rapidly emerged as a facile and efficient platform for genome editing. Here, we use a human cell-based reporter assay to characterize off-target cleavage of CRISPR-associated (Cas)9-based RGNs. We find that single and double mismatches are tolerated to varying degrees depending on their position along the guide RNA (gRNA)-DNA interface. We also readily detected off-target alterations induced by four out of six RGNs targeted to endogenous loci in human cells by examination of partially mismatched sites. The off-target sites we identified harbored up to five mismatches and many were mutagenized with frequencies comparable to (or higher than) those observed at the intended on-target site. Our work demonstrates that RGNs can be highly active even with imperfectly matched RNA-DNA interfaces in human cells, a finding that might confound their use in research and therapeutic 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.