Genome-Editing Technologies: Principles and Applications

被引：190
作者：
Gaj, Thomas [1 ]
Sirk, Shannon J. [2 ]
Shui, Sai-lan [3 ]
Liu, Jia [3 ]
机构：
[1] Univ Calif Berkeley, Dept Bioengn, Berkeley, CA 94720 USA
[2] Stanford Univ, Dept Chem Engn, Stanford, CA 94305 USA
[3] ShanghaiTech Univ, Shanghai Inst Adv Immunochem Studies, Shanghai, Peoples R China
来源：
COLD SPRING HARBOR PERSPECTIVES IN BIOLOGY | 2016年 / 8卷 / 12期
基金：
美国国家卫生研究院;
关键词：
ZINC-FINGER NUCLEASES; CONTROLLING GENE-EXPRESSION; HOMOLOGY-DIRECTED REPAIR; DOUBLE-STRAND BREAKS; WIDE CRISPR SCREEN; HUMAN-CELLS; DNA-RECOGNITION; TRANSCRIPTION FACTORS; CRYSTAL-STRUCTURE; TAL EFFECTORS;
D O I：
10.1101/cshperspect.a023754
中图分类号：
Q2 [细胞生物学];
学科分类号：
071009 ; 090102 ;
摘要：
Targeted nucleases have provided researchers with the ability to manipulate virtually any genomic sequence, enabling the facile creation of isogenic cell lines and animal models for the study of human disease, and promoting exciting new possibilities for human gene therapy. Here we review three foundational technologies-clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription activator-like effector nucleases (TALENs), and zinc-finger nucleases (ZFNs). We discuss the engineering advances that facilitated their development and highlight several achievements in genome engineering that were made possible by these tools. We also consider artificial transcription factors, illustrating how this technology can complement targeted nucleases for synthetic biology and gene therapy.
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页数：20
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