Isolation of single-base genome-edited human iPS cells without antibiotic selection

被引：197

作者：

Miyaoka, Yuichiro ^{[1
]}

Chan, Amanda H. ^{[1
]}

Judge, Luke M. ^{[1
,2
]}

Yoo, Jennie ^{[1
]}

Huang, Miller ^{[3
]}

Nguyen, Trieu D. ^{[1
]}

Lizarraga, Paweena P. ^{[1
]}

So, Po-Lin ^{[1
]}

Conklin, Bruce R. ^{[1
,4
,5
]}

机构：

[1] Gladstone Inst Cardiovasc Dis, San Francisco, CA 94141 USA

[2] Univ Calif San Francisco, Dept Pediat, San Francisco, CA USA

[3] Univ Calif San Francisco, Dept Neurol, San Francisco, CA USA

[4] Univ Calif San Francisco, Dept Med, San Francisco, CA USA

[5] Univ Calif San Francisco, Dept Mol & Cellular Pharmacol, San Francisco, CA USA

来源：

NATURE METHODS | 2014年 / 11卷 / 03期

基金：

日本学术振兴会; 美国国家卫生研究院;

关键词：

PLURIPOTENT STEM-CELLS; ZINC-FINGER TARGETER; TALEN; DESIGN; CAS9; SPECIFICITY; MUTATIONS; NUCLEASES; SYSTEM; ZIFIT;

D O I：

10.1038/nmeth.2840

中图分类号：

Q5 [生物化学];

学科分类号：

071010 ; 081704 ;

摘要：

Precise editing of human genomes in pluripotent stem cells by homology-driven repair of targeted nuclease-induced cleavage has been hindered by the difficulty of isolating rare clones. We developed an efficient method to capture rare mutational events, enabling isolation of mutant lines with single-base substitutions without antibiotic selection. This method facilitates efficient induction or reversion of mutations associated with human disease in isogenic human induced pluripotent stem cells.

引用

页码：291 / U345

页数：7

共 30 条

[1] In vivo genome editing using a high-efficiency TALEN system [J].

Bedell, Victoria M. ;

Wang, Ying ;

Campbell, Jarryd M. ;

Poshusta, Tanya L. ;

Starker, Colby G. ;

Krug, Randall G., II ;

Tan, Wenfang ;

Penheiter, Sumedha G. ;

Ma, Alvin C. ;

Leung, Anskar Y. H. ;

Fahrenkrug, Scott C. ;

Carlson, Daniel F. ;

Voytas, Daniel F. ;

Clark, Karl J. ;

Essner, Jeffrey J. ;

Ekker, Stephen C. .

NATURE, 2012, 491 (7422) :114-U133

[2] Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting [J].

Cermak, Tomas ;

Doyle, Erin L. ;

Christian, Michelle ;

Wang, Li ;

Zhang, Yong ;

Schmidt, Clarice ;

Baller, Joshua A. ;

Somia, Nikunj V. ;

Bogdanove, Adam J. ;

Voytas, Daniel F. .

NUCLEIC ACIDS RESEARCH, 2011, 39 (12) :e82

[3] High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases [J].

Chen, Fuqiang ;

Pruett-Miller, Shondra M. ;

Huang, Yuping ;

Gjoka, Monika ;

Duda, Katarzyna ;

Taunton, Jack ;

Collingwood, Trevor N. ;

Frodin, Morten ;

Davis, Gregory D. .

NATURE METHODS, 2011, 8 (09) :753-U96

[4] Targeting G with TAL Effectors: A Comparison of Activities of TALENs Constructed with NN and NK Repeat Variable Di-Residues [J].

Christian, Michelle L. ;

Demorest, Zachary L. ;

Starker, Colby G. ;

Osborn, Mark J. ;

Nyquist, Michael D. ;

Zhang, Yong ;

Carlson, Daniel F. ;

Bradley, Philip ;

Bogdanove, Adam J. ;

Voytas, Daniel F. .

PLOS ONE, 2012, 7 (09)

[5] Multiplex Genome Engineering Using CRISPR/Cas Systems [J].

Cong, Le ;

Ran, F. Ann ;

Cox, David ;

Lin, Shuailiang ;

Barretto, Robert ;

Habib, Naomi ;

Hsu, Patrick D. ;

Wu, Xuebing ;

Jiang, Wenyan ;

Marraffini, Luciano A. ;

Zhang, Feng .

SCIENCE, 2013, 339 (6121) :819-823

[6] Comprehensive interrogation of natural TALE DNA-binding modules and transcriptional repressor domains [J].

Cong, Le ;

Zhou, Ruhong ;

Kuo, Yu-chi ;

Cunniff, Margaret ;

Zhang, Feng .

NATURE COMMUNICATIONS, 2012, 3

[7] A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models [J].

Ding, Qiurong ;

Lee, Youn-Kyoung ;

Schaefer, Esperance A. K. ;

Peters, Derek T. ;

Veres, Adrian ;

Kim, Kevin ;

Kuperwasser, Nicolas ;

Motola, Daniel L. ;

Meissner, Torsten B. ;

Hendriks, William T. ;

Trevisan, Marta ;

Gupta, Rajat M. ;

Moisan, Annie ;

Banks, Eric ;

Friesen, Max ;

Schinzel, Robert T. ;

Xia, Fang ;

Tang, Alexander ;

Xia, Yulei ;

Figueroa, Emmanuel ;

Wann, Amy ;

Ahfeldt, Tim ;

Daheron, Laurence ;

Zhang, Feng ;

Rubin, Lee L. ;

Peng, Lee F. ;

Chung, Raymond T. ;

Musunuru, Kiran ;

Cowan, Chad A. .

CELL STEM CELL, 2013, 12 (02) :238-251

[8] TAL Effector-Nucleotide Targeter (TALE-NT) 2.0: tools for TAL effector design and target prediction [J].

Doyle, Erin L. ;

Booher, Nicholas J. ;

Standage, Daniel S. ;

Voytas, Daniel F. ;

Brendel, Volker P. ;

VanDyk, John K. ;

Bogdanove, Adam J. .

NUCLEIC ACIDS RESEARCH, 2012, 40 (W1) :W117-W122

[9] High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells [J].

Fu, Yanfang ;

Foden, Jennifer A. ;

Khayter, Cyd ;

Maeder, Morgan L. ;

Reyon, Deepak ;

Joung, J. Keith ;

Sander, Jeffry D. .

NATURE BIOTECHNOLOGY, 2013, 31 (09) :822-+

[10] ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering [J].

Gaj, Thomas ;

Gersbach, Charles A. ;

Barbas, Carlos F., III .

TRENDS IN BIOTECHNOLOGY, 2013, 31 (07) :397-405

← 1 2 3 →