CRISPR/Cas9 Genome-Editing System in Human Stem Cells: Current Status and Future Prospects

被引：70
|
作者：
Zhang, Zhao [1 ]
Zhang, Yuelin [1 ]
Gao, Fei [1 ]
Han, Shuo [1 ]
Cheah, Kathryn S. [3 ]
Tse, Hung-Fat [1 ,2 ]
Lian, Qizhou [1 ,2 ,3 ]
机构：
[1] Univ Hong Kong, Dept Med, Hong Kong, Hong Kong, Peoples R China
[2] Univ Hong Kong, Shenzhen Inst Res & Innovat, Shenzhen, Peoples R China
[3] Univ Hong Kong, Sch Biomed Sci, Hong Kong, Hong Kong, Peoples R China
来源：
MOLECULAR THERAPY-NUCLEIC ACIDS | 2017年 / 9卷
关键词：
HOMOLOGY-DIRECTED REPAIR; SEQUENCE-SPECIFIC CONTROL; HUMAN HEMATOPOIETIC STEM; STRAND BREAK REPAIR; CRISPR-CAS9; NUCLEASES; GENE CORRECTION; HIV-1; INFECTION; DYSTROPHIN GENE; IMMUNE CELLS; BETA-GLOBIN;
D O I：
10.1016/j.omtn.2017.09.009
中图分类号：
R-3 [医学研究方法]; R3 [基础医学];
学科分类号：
1001 ;
摘要：
Genome-editing involves the insertion, deletion, or replacement of DNA in the genome of a living organism using "molecular scissors." Traditional genome editing with engineered nucleases for human stem cells is limited by its low efficiency, high cost, and poor specificity. The CRISPR system has recently emerged as a powerful gene manipulation technique with advantages of high editing efficiency and low cost. Although this technique offers huge potential for gene manipulation in various organisms ranging from prokaryotes to higher mammals, there remain many challenges in human stem cell research. In this review, we highlight the basic biology and application of the CRISPR/Cas9 system in current human stem cell research, discuss its advantages and challenges, and debate the future prospects for human stem cells in regenerative medicine.
引用
收藏
页码：230 / 241
页数：12
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