文献类型：期刊文献

英文题名：Characterization of Brevibacillus laterosporus Cas9 (BlatCas9) for Mammalian Genome Editing

作者：Gao, Ning[1,2] Zhang, Chengdong[2] Hu, Ziying[2] Li, Miaomiao[2] Wei, Jingjing[2] Wang, Yongming[2,3] Liu, Huihui[1]

第一作者：Gao, Ning

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

机构：[1]Chinese Acad Forestry, Expt Ctr Forestry North China, Beijing, Peoples R China;[2]Fudan Univ, State Key Lab Genet Engn, Sch Life Sci, Zhongshan Hosp,Obstet & Gynecol Hosp, Shanghai, Peoples R China;[3]Shanghai Engn Res Ctr Ind Microorganisms, Shanghai, Peoples R China

年份：2020

卷号：8

外文期刊名：FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY

收录：;Scopus(收录号：2-s2.0-85094938727);WOS:【SCI-EXPANDED(收录号:WOS:000583274400001)】；

基金：This work was supported by grants from the Fundamental fund of CAF (CAFYBB2017MA018) and the National Natural Science Foundation of China (31700571 and 81870199), the Foundation for Innovative Research Group of the National Natural Science Foundation of China (31521003).

语种：英文

外文关键词：CRISPR; Cas9; genome editing; compact; aav; gene therapy

摘要：Compact CRISPR/Cas9 systems that can be delivered by AAV for in vivo genome editing hold great promise for clinical applications. Brevibacillus laterosporus Cas9 (BlatCas9) is a compact Cas9 nuclease that has been identified for plant genome editing. Here, we characterize BlatCas9 as an alternative tool for mammalian genome editing. We demonstrate that BlatCas9 prefers a N4CNAA protospacer adjacent motif (PAM), but N4C PAM is also editable in mammalian cells. We next demonstrate that BlatCas9 enables genome editing in a variety of cell types. Furthermore, BlatCas9 can be packaged into AAV for genome editing. Finally, we characterize the specificity of BlatCas9. In summary, BlatCas9 offers an alternative tool for both basic research and clinical applications.