CRISPR/Cas9-mediated correction of human genetic disease

• 作 者: Ke Men Xingmei Duan Zhiyao He
• 机 构: StateKeyLaboratoryofBiotherapyandCancerCenter/CollaborativeInnovationCenterforBiotherapy,Individuali
• 出 处: 中国科学：生命科学英文版
• 发表日期: 2017年5期
• 关键词: 介导 人类 遗传病 非同源末端连接 序列特异性 DNA修复 校正 临床治疗 CRISPR/Cas9 genome editing genetic disea
• 基金项目: supported by the National Natural Science Foundation （NSFC81502677, NSFC81602699, NSFC81123003）, the National Key Research and Development Program of China （2016YFA0201402）, the Key Technologies R ＆ D

The clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated(Cas) protein 9 system(CRISPR/Cas9) provides a powerful tool for targeted genetic editing. Directed by programmable sequence-specific RNAs,this system introduces cleava