1. CRISPR–Cas9 gene editing induced complex on-target outcomes in human cells.
- Author
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Wen, Wei and Zhang, Xiao-Bing
- Subjects
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GENOME editing , *CRISPRS , *GENE rearrangement , *GENE therapy , *QUALITY control , *ANAPLASTIC lymphoma kinase , *DELETION mutation - Abstract
• Unwanted on-target mutation occurs after CRISPR–Cas9 cleavage. • Assessment of comprehensive on-target outcomes is necessary. • Clinical genomic engineering requires quality controls to address safety concerns. CRISPR–Cas9 is a powerful tool for editing the genome and holds great promise for gene therapy applications. Initial concerns of gene engineering focus on off-target effects. However, in addition to short indel mutations (often <50 bp), an increasing number of studies have revealed complex on-target results after double-strand break repair by CRISPR–Cas9, such as large deletions, gene rearrangement, and loss of heterozygosity. These unintended mutations are potential safety concerns in clinical gene editing. Here, in this review, we summarize the significant findings of CRISPR–Cas9-induced on-target deleterious outcomes and discuss putative ways to achieve safe gene therapy. [ABSTRACT FROM AUTHOR]
- Published
- 2022
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