机构：[1]Department of Infectious Disease, Shenzhen People’s Hospital, The Second Clinical Medical College of Jinan University, The First Affiliated Hospital of Southern University of Science and Technology, Shenzhen 518020, China深圳市康宁医院深圳市人民医院深圳医学信息中心中国医科大学附属盛京医院中国医科大学盛京医院[2]Yunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, Yunnan 650500, China[3]Division of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, Ohio 43210, USA
The CRISPR (clustered regularly interspaced short palindromic repeats)-based genome editing technology is an emerging RNA-guided nuclease system initially identified from the microbial adaptive immune systems. In recent years, the CRISPR system has been reprogrammed to target specific regions of the eukaryotic genome and has become a powerful tool for genetic engineering. Researchers have explored many approaches to improve the genome editing activity of the CRISPR-Cas system and deliver its components both ex vivo and in vivo. Moreover, these strategies have been applied to genome editing in preclinical research and clinical trials. In this review, we focus on representative strategies for regulation and delivery of the CRISPR-Cas system, and outline current therapeutic applications in their clinical translation.
基金：
National Key Research and Development Program [2016YFA0101401, R35GM119679]; National Institute of General Medical SciencesUnited States Department of Health & Human ServicesNational Institutes of Health (NIH) - USANIH National Institute of General Medical Sciences (NIGMS)
第一作者机构：[1]Department of Infectious Disease, Shenzhen People’s Hospital, The Second Clinical Medical College of Jinan University, The First Affiliated Hospital of Southern University of Science and Technology, Shenzhen 518020, China
通讯作者：
推荐引用方式(GB/T 7714)：
Li Bin,Niu Yuyu,Ji Weizhi,et al.Strategies for the CRISPR-Based Therapeutics[J].TRENDS IN PHARMACOLOGICAL SCIENCES.2020,41(1):55-65.doi:10.1016/j.tips.2019.11.006.
APA：
Li, Bin,Niu, Yuyu,Ji, Weizhi&Dong, Yizhou.(2020).Strategies for the CRISPR-Based Therapeutics.TRENDS IN PHARMACOLOGICAL SCIENCES,41,(1)
MLA：
Li, Bin,et al."Strategies for the CRISPR-Based Therapeutics".TRENDS IN PHARMACOLOGICAL SCIENCES 41..1(2020):55-65
：
医学