机构：[1]State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, Sichuan University and Collaborative Innovation Center , Chengdu, China.四川大学华西医院[2]Department of Pharmacy, and Cancer Center, West China Hospital, Sichuan University and Collaborative Innovation Center , Chengdu, China.四川大学华西医院[3]Yunnan Key Laboratory of Primate Biomedicine Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology , Kunming, China
Editing of the genome to correct disease-causing mutations is a promising approach for the treatment of human diseases. Recent advances in the development of programmable nuclease-based genome editing tools have substantially improved the ability to make precise changes in the human genome. Genome editing technologies are already being used to correct genetic mutations in affected tissues and cells to treat diseases that are refractory to traditional gene therapies. Chinese scientists have made remarkable breakthroughs in the field of therapeutic genome editing, particularly with the first clinical trial involving the clustered regularly interspaced short palindromic repeats-caspase 9 system that began in China. Herein, current progress toward developing programmable nuclease-based gene therapies is introduced, as well as future prospects and challenges in China.
语种：
外文
PubmedID：
中科院(CAS)分区：
出版当年[2018]版：
大类|2 区医学
小类|2 区生物工程与应用微生物2 区遗传学2 区医学：研究与实验
最新[2023]版：
大类|3 区医学
小类|2 区生物工程与应用微生物3 区遗传学3 区医学：研究与实验
第一作者：
第一作者机构：[1]State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, Sichuan University and Collaborative Innovation Center , Chengdu, China.[*1]State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, Sichuan University and Collaborative Innovation Center, Chengdu 610041, China, Ke-yuan Road 4, No. 1, Gao-peng Street, Chengdu, Sichuan, 610041, China
共同第一作者：
通讯作者：
通讯机构：[1]State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, Sichuan University and Collaborative Innovation Center , Chengdu, China.[*1]State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, Sichuan University and Collaborative Innovation Center, Chengdu 610041, China, Ke-yuan Road 4, No. 1, Gao-peng Street, Chengdu, Sichuan, 610041, China
推荐引用方式(GB/T 7714)：
Yang Yang,Wang Qingnan,Li Qian,et al.Recent Advances in Therapeutic Genome Editing in China.[J].Human gene therapy.2018,29(2):136-145.doi:10.1089/hum.2017.210.
APA：
Yang Yang,Wang Qingnan,Li Qian,Men Ke,He Zhiyao...&Wei Yuquan.(2018).Recent Advances in Therapeutic Genome Editing in China..Human gene therapy,29,(2)
MLA：
Yang Yang,et al."Recent Advances in Therapeutic Genome Editing in China.".Human gene therapy 29..2(2018):136-145
