文献类型：期刊文献

英文题名：A compact Cas9 ortholog from Staphylococcus Auricularis (SauriCas9) expands the DNA targeting scope

作者：Hu, Ziying[1] Wang, Shuai[1] Zhang, Chengdong[1] Gao, Ning[1] Li, Miaomiao[1] Wang, Deqian[1] Wang, Daqi[1] Liu, Dong[2] Liu, Huihui[3] Ong, Sang-Ging[4,5] Wang, Hongyan[1] Wang, Yongming[1,2,6]

第一作者：Hu, Ziying

通信作者：Wang, YM[1];Wang, YM[2];Wang, YM[3]

机构：[1]Fudan Univ, Sch Life Sci, Zhongshan Hosp, Obstet & Gynecol Hosp,State Key Lab Genet Engn, Shanghai, Peoples R China;[2]Nantong Univ, Coinnovat Ctr Neuroregenerat, Jiangsu Key Lab Neuroregenerat, Sch Life Sci, Nantong, Peoples R China;[3]Chinese Acad Forestry, Expt Ctr Forestry North China, Beijing, Peoples R China;[4]Univ Illinois, Coll Med, Dept Pharmacol, Chicago, IL 60612 USA;[5]Univ Illinois, Coll Med, Dept Med, Div Cardiol, Chicago, IL USA;[6]Shanghai Engn Res Ctr Ind Microorganisms, Shanghai, Peoples R China

年份：2020

卷号：18

期号：3

外文期刊名：PLOS BIOLOGY

收录：;Scopus(收录号：2-s2.0-85083536769);WOS:【SCI-EXPANDED(收录号:WOS:000558340000011)】；

基金：HL was supported by grants from the Fundamental fund of CAF (CAFYBB2017MA018) and the National Natural Science Foundation of China (31700571); YW was supported by grants from the National Natural Science Foundation of China (81870199), the Foundation for Innovative Research Group of the National Natural Science Foundation of China (31521003), the State Key Laboratory Opening Program SKLGE1809 and 111 project (B13016); SO was supported by grants from the National Institutes of Health R00HL130416. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.

语种：英文

摘要：Compact CRISPR/Cas9 systems that can be packaged into an adeno-associated virus (AAV) hold great promise for gene therapy. Unfortunately, currently available small Cas9 nucleases either display low activity or require a long protospacer adjacent motif (PAM) sequence, limiting their extensive applications. Here, we screened a panel of Cas9 nucleases and identified a small Cas9 ortholog from Staphylococcus auricularis (SauriCas9), which recognizes a simple NNGG PAM, displays high activity for genome editing, and is compact enough to be packaged into an AAV for genome editing. Moreover, the conversion of adenine and cytosine bases can be achieved by fusing SauriCas9 to the cytidine and adenine deaminase. Therefore, SauriCas9 holds great potential for both basic research and clinical applications.