Genetic engineering of human pluripotent cells using TALE nucleases

被引:869
作者
Hockemeyer, Dirk [1 ]
Wang, Haoyi [1 ]
Kiani, Samira [1 ]
Lai, Christine S. [1 ,2 ]
Gao, Qing [1 ]
Cassady, John P. [1 ,2 ]
Cost, Gregory J. [3 ]
Zhang, Lei [3 ]
Santiago, Yolanda [3 ]
Miller, Jeffrey C. [3 ]
Zeitler, Bryan [3 ]
Cherone, Jennifer M. [3 ]
Meng, Xiangdong [3 ]
Hinkley, Sarah J. [3 ]
Rebar, Edward J. [3 ]
Gregory, Philip D. [3 ]
Urnov, Fyodor D. [3 ]
Jaenisch, Rudolf [1 ,2 ]
机构
[1] Whitehead Inst Biomed Res, Cambridge, MA USA
[2] MIT, Dept Biol, Cambridge, MA USA
[3] Sangamo BioSci Inc, Richmond, CA USA
基金
美国国家卫生研究院;
关键词
ZINC-FINGER NUCLEASES; EMBRYONIC STEM-CELLS; III EFFECTORS;
D O I
10.1038/nbt.1927
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
Targeted genetic engineering of human pluripotent cells is a prerequisite for exploiting their full potential. Such genetic manipulations can be achieved using site-specific nucleases. Here we engineered transcription activator-like effector nucleases (TALENs) for five distinct genomic loci. At all loci tested we obtained human embryonic stem cell (ESC) and induced pluripotent stem cell (iPSC) clones carrying transgenic cassettes solely at the TALEN-specified location. Our data suggest that TALENs employing the specific architectures described here mediate site-specific genome modification in human pluripotent cells with similar efficiency and precision as do zinc-finger nucleases (ZFNs).
引用
收藏
页码:731 / 734
页数:4
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