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 Z. Wang S. Zhang C. Gao N. Li M. Wang D. Wang D. Liu D. Liu H. Ong S.-G. Wang H. Wang Y.

机构：[1]Obstetrics and Gynecology Hospital, State Key Laboratory of Genetic Engineering, School of Life Sciences, Zhongshan Hospital, Fudan University, Shanghai, China;[2]School of Life Sciences, Co-innovation Center of Neuroregeneration, Jiangsu Key Laboratory of Neuroregeneration, Nantong University, Nantong, China;[3]Experimental Center of Forestry in North China, Chinese Academy of Forestry, Beijing, China;[4]Department of Pharmacology, University of Illinois College of Medicine, Chicago, IL, United States;[5]Division of Cardiology, Department of Medicine, University of Illinois College of Medicine, Chicago, IL, United States;[6]Shanghai Engineering Research Center of Industrial Microorganisms, Shanghai, 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)

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. ? 2020 Hu et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.