同源定向修复
RAD52
清脆的
基因组编辑
Cas9
生物
DNA修复
异位表达
DNA
诱导多能干细胞
细胞生物学
基因
遗传学
核苷酸切除修复
计算生物学
雷达51
胚胎干细胞
作者
Bruna S. Paulsen,Pankaj Kumar Mandal,Richard L. Frock,Bariş Boyraz,Rachita Yadav,Srigokul Upadhyayula,Paula Gutierrez‐Martinez,Wataru Ebina,Anders Fasth,Tomas Kirchhausen,Michael E. Talkowski,Suneet Agarwal,Frederick W. Alt,Derrick J. Rossi
标识
DOI:10.1038/s41551-017-0145-2
摘要
Gene disruption by clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9) is highly efficient and relies on the error-prone non-homologous end-joining pathway. Conversely, precise gene editing requires homology-directed repair (HDR), which occurs at a lower frequency than non-homologous end-joining in mammalian cells. Here, by testing whether manipulation of DNA repair factors improves HDR efficacy, we show that transient ectopic co-expression of RAD52 and a dominant-negative form of tumour protein p53-binding protein 1 (dn53BP1) synergize to enable efficient HDR using a single-stranded oligonucleotide DNA donor template at multiple loci in human cells, including patient-derived induced pluripotent stem cells. Co-expression of RAD52 and dn53BP1 improves multiplexed HDR-mediated editing, whereas expression of RAD52 alone enhances HDR with Cas9 nickase. Our data show that the frequency of non-homologous end-joining-mediated double-strand break repair in the presence of these two factors is not suppressed and suggest that dn53BP1 competitively antagonizes 53BP1 to augment HDR in combination with RAD52. Importantly, co-expression of RAD52 and dn53BP1 does not alter Cas9 off-target activity. These findings support the use of RAD52 and dn53BP1 co-expression to overcome bottlenecks that limit HDR in precision genome editing.
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