CRISPR/Cas9 system： a powerful technology for in vivo and ex vivo gene therapy

作     者：Xiaohui Zhang Liren Wang Mingyao Liu Dali Li 

作者机构：Shanghai Key Laboratory of Regulatory Biology institute of Biomedical Sciences and School of Life Sciences East China Normal University Shanghai 200241 China 

出 版 物：《Science China(Life Sciences)》 (中国科学（生命科学英文版）)

年 卷 期：2017年第60卷第5期

页      面：468-475页

核心收录：

学科分类：1002[医学-临床医学] 1010[医学-医学技术（可授医学、理学学位）] 100215[医学-康复医学与理疗学] 10[医学] 

基　　金：supported by the National Natural Science Foundation of China (31371455, 31171318 to Dali Li, 81330049 to Mingyao Liu) the Science and Technology Commission of Shanghai Municipality (14140900300 to Dali Li) 

主　　题：CRISPR/Cas9 gene therapy in vivo ex vivo 

摘      要：CRISPR/Cas9 is a versatile genome-editing tool which is widely used for modifying the genome of both prokaryotic and eukaryotic organisms for basic research and applications. An increasing number of reports have demonstrated that CRISPR/Cas9-mediated genome editing is a powerful technology for gene therapy. Here, we review the recent advances in CRISPR/Cas9-mediated gene therapy in animal models via different strategies and discuss the challenges as well as future prospects.