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论文题目: HIT-Cas9: a CRISPR/Cas9 Genome Editing Device Under Tight and Effective Drug Control
论文题目英文:
作者: 赵晨①,赵迎泽①,张竞方①,卢佳,陈丽,张月,Yue Ying,#Jun-Jun Xu,Shi-Xian Wei,王宇*
论文出处:
年: 2018
卷:
期: DOI: 10.1016/j.omtn.2018.08.022
页:
联系作者: 王宇
发表期刊: Molecular Therapy - Nucleic Acids
ISSN:
第一作者所在部门:
收录类别:
论文链接: https://www.sciencedirect.com/science/article/pii/S2162253118302373
影响因子:
摘要: The CRISPR/Cas9 (clustered, regularly interspaced, short palindromic repeats /CRISPR associated protein9) enabled efficient gene editing in an easy and programmable manner. Controlling its activity in greater precision is desired for biomedical research and potential therapeutic translation. Here we engrafted the CRISPR/Cas9 system with a mutated human estrogen receptor (ERT2), which renders it 4-hydroxytamoxifen (4-OHT) inducible for the access of genome, and a nuclear export signal (NES), which lowers the background activity. Tight and efficient drug inducible genome editing was achieved across several human cell types, including embryonic stem cells (ESCs) and mesenchymal stem cells (MSCs), upon vigorous optimization. Optimized terminal device, which we named Hybrid drug Inducible CRISPR/Cas9 Technology (HIT-Cas9), delivered advantageous performances over several existing designs. Such architecture was also successfully applied to an orthogonal Cas9. The HIT-Cas9 system developed in this study will find broad utility in controlled editing of potentially any genomic loci.
英文摘要:
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