Cas9
转基因
清脆的
基因组编辑
同源重组
生物
基因座(遗传学)
同源定向修复
增强子
融合蛋白
计算生物学
细胞生物学
遗传学
基因
重组DNA
DNA修复
基因表达
DNA错配修复
作者
Maud Charpentier,Ahmed Khedher,Séverine Ménoret,A. Brion,Khadija Lamribet,Elodie Dardillac,Charlotte Boix,L. Perrouault,Laurent Tesson,Sylvain Geny,Anne De Cian,Jean-Michiel Itier,Ignacio Anegón,Bernard S. López,Carine Giovannangeli,Jean‐Paul Concordet
标识
DOI:10.1038/s41467-018-03475-7
摘要
In genome editing with CRISPR-Cas9, transgene integration often remains challenging. Here, we present an approach for increasing the efficiency of transgene integration by homology-dependent repair (HDR). CtIP, a key protein in early steps of homologous recombination, is fused to Cas9 and stimulates transgene integration by HDR at the human AAVS1 safe harbor locus. A minimal N-terminal fragment of CtIP, designated HE for HDR enhancer, is sufficient to stimulate HDR and this depends on CDK phosphorylation sites and the multimerization domain essential for CtIP activity in homologous recombination. HDR stimulation by Cas9-HE, however, depends on the guide RNA used, a limitation that may be overcome by testing multiple guides to the locus of interest. The Cas9-HE fusion is simple to use and allows obtaining twofold or more efficient transgene integration than that with Cas9 in several experimental systems, including human cell lines, iPS cells, and rat zygotes.
科研通智能强力驱动
Strongly Powered by AbleSci AI