Efficient Precision Genome Editing in iPSCs via Genetic Co-targeting with Selection

被引：25

作者：

Mitzelfelt, Katie A. ^{[1
]}

McDermott-Roe, Chris ^{[2
,3
]}

Grzybowski, Michael N. ^{[2
,3
]}

Marquez, Maribel ^{[2
,3
]}

Kuo, Chieh-Ti ^{[2
]}

Riedel, Michael ^{[4
]}

Lai, Shuping ^{[2
]}

Choi, Melinda J. ^{[2
]}

Kolander, Kurt D. ^{[2
]}

Helbling, Daniel ^{[5
]}

Dimmock, David P. ^{[5
]}

Battle, Michele A. ^{[6
]}

Jou, Chuanchau J. ^{[7
,8
]}

Tristani-Firouzi, Martin ^{[7
,8
]}

Verbsky, James W. ^{[9
]}

Benjamin, Ivor J. ^{[1
,2
]}

Geurts, Aron M. ^{[2
,3
]}

机构：

[1] Univ Utah, Dept Biochem, Salt Lake City, UT 84112 USA

[2] Med Coll Wisconsin, Cardiovasc Ctr, Milwaukee, WI 53226 USA

[3] Med Coll Wisconsin, Dept Physiol, Milwaukee, WI 53226 USA

[4] PharmaCell, NL-6229 Maastricht, Netherlands

[5] Med Coll Wisconsin, Human Mol Genet Ctr, Dept Pediat, Div Genet, Milwaukee, WI 53226 USA

[6] Med Coll Wisconsin, Dept Cell Biol Neurobiol & Anat, Milwaukee, WI 53226 USA

[7] Univ Utah, Nora Eccles Harrison CVRTI, Sch Med, Salt Lake City, UT 84112 USA

[8] Univ Utah, Sch Med, Div Pediat Cardiol, Salt Lake City, UT 83113 USA

[9] Med Coll Wisconsin, Sect Quantitat Hlth Sci, Dept Pediat, Milwaukee, WI 53226 USA

来源：

STEM CELL REPORTS | 2017年 / 8卷 / 03期

关键词：

PLURIPOTENT STEM-CELLS; CAENORHABDITIS-ELEGANS; CRISPR-CAS9; NUCLEASES; CARDIOMYOCYTES; GENERATION; SYSTEMS; REPAIR;

D O I：

10.1016/j.stemcr.2017.01.021

中图分类号：

Q813 [细胞工程];

学科分类号：

摘要：

Genome editing in induced pluripotent stem cells is currently hampered by the laborious and expensive nature of identifying homology-directed repair (HDR)-modified cells. We present an approach where isolation of cells bearing a selectable, HDR-mediated editing event at one locus enriches for HDR-mediated edits at additional loci. This strategy, called co-targeting with selection, improves the probability of isolating cells bearing HDR-mediated variants and accelerates the production of disease models.

引用

页码：491 / 499

页数：9

共 33 条

[1] Efficient Marker-Free Recovery of Custom Genetic Modifications with CRISPR/Cas9 in Caenorhabditis elegans [J].

Arribere, Joshua A. ;

Bell, Ryan T. ;

Fu, Becky X. H. ;

Artiles, Karen L. ;

Hartman, Phil S. ;

Fire, Andrew Z. .

GENETICS, 2014, 198 (03) :837-U842

[2] CRISPR-Cas Systems in Bacteria and Archaea: Versatile Small RNAs for Adaptive Defense and Regulation [J].

Bhaya, Devaki ;

Davison, Michelle ;

Barrangou, Rodolphe .

ANNUAL REVIEW OF GENETICS, VOL 45, 2011, 45 :273-297

[3] Trimmomatic: a flexible trimmer for Illumina sequence data [J].

Bolger, Anthony M. ;

Lohse, Marc ;

Usadel, Bjoern .

BIOINFORMATICS, 2014, 30 (15) :2114-2120

[4] 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

[5] Enhanced Efficiency of Human Pluripotent Stem Cell Genome Editing through Replacing TALENs with CRISPRs [J].

Ding, Qiurong ;

Regan, Stephanie N. ;

Xia, Yulei ;

Oostrom, Leonie A. ;

Cowan, Chad A. ;

Musunuru, Kiran .

CELL STEM CELL, 2013, 12 (04) :393-394

[6] 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

[7] Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9 [J].

Doench, John G. ;

Fusi, Nicolo ;

Sullender, Meagan ;

Hegde, Mudra ;

Vaimberg, Emma W. ;

Donovan, Katherine F. ;

Smith, Ian ;

Tothova, Zuzana ;

Wilen, Craig ;

Orchard, Robert ;

Virgin, Herbert W. ;

Listgarten, Jennifer ;

Root, David E. .

NATURE BIOTECHNOLOGY, 2016, 34 (02) :184-+

[8]

Elliott DA, 2011, NAT METHODS, V8, P1037, DOI [10.1038/nmeth.1740, 10.1038/NMETH.1740]

[9] Single-Step Generation of Conditional Knockout Mouse Embryonic Stem Cells [J].

Flemr, Matyas ;

Buehler, Marc .

CELL REPORTS, 2015, 12 (04) :709-716

[10] Knockout Rats via Embryo Microinjection of Zinc-Finger Nucleases [J].

Geurts, Aron M. ;

Cost, Gregory J. ;

Freyvert, Yevgeniy ;

Zeitler, Bryan ;

Miller, Jeffrey C. ;

Choi, Vivian M. ;

Jenkins, Shirin S. ;

Wood, Adam ;

Cui, Xiaoxia ;

Meng, Xiangdong ;

Vincent, Anna ;

Lam, Stephen ;

Michalkiewicz, Mieczyslaw ;

Schilling, Rebecca ;

Foeckler, Jamie ;

Kalloway, Shawn ;

Weiler, Hartmut ;

Menoret, Severine ;

Anegon, Ignacio ;

Davis, Gregory D. ;

Zhang, Lei ;

Rebar, Edward J. ;

Gregory, Philip D. ;

Urnov, Fyodor D. ;

Jacob, Howard J. ;

Buelow, Roland .

SCIENCE, 2009, 325 (5939) :433-433

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