Targeted genome editing in pluripotent stem cells using zinc-finger nucleases

被引：19

作者：

Bobis-Wozowicz, Sylwia ^{[1
]}

Osiak, Anna ^{[1
]}

Rahman, Shamim H. ^{[1
]}

Cathomen, Toni ^{[1
]}

机构：

[1] Hannover Med Sch, Dept Expt Hematol, D-30625 Hannover, Germany

来源：

METHODS | 2011年 / 53卷 / 04期

关键词：

Zinc-finger nuclease; Genome engineering; Gene targeting; Gene disruption; Embryonic stem cells; HOMOLOGOUS RECOMBINATION; GENE-THERAPY; T-CELLS; TRANSCRIPTION FACTORS; RESTRICTION ENZYMES; FUNCTIONAL-ANALYSIS; CHIMERIC NUCLEASES; MAJOR DETERMINANT; MAMMALIAN GENOME; GENERATION;

D O I：

10.1016/j.ymeth.2010.12.019

中图分类号：

Q5 [生物化学];

学科分类号：

071010 ; 081704 ;

摘要：

Zinc-finger nucleases (ZFNs) are designer nucleases capable of cleaving a prespecified target DNA within complex genomes. ZFNs consist of a non-specific endonuclease domain fused to an engineered DNA-binding domain that tethers the nuclease activity to the chosen chromosomal site. The endonuclease-induced DNA double strand break triggers a cellular DNA damage response, resulting in double strand break repair by either accurate homologous recombination (HR) or error-prone non-homologous end-joining (NHEJ). Thus, ZFNs are powerful tools for targeted genome engineering in a variety of mammalian cell types, including embryonic (ESCs) and induced pluripotent stem cells (iPSCs). As a paradigm for genome editing in pluripotent stem cells, we describe the use of ZFNs in murine ESCs for generating knockout alleles by NHEJ without selection or by HR employing different selection schemes. (C) 2011 Elsevier Inc. All rights reserved.

引用

页码：339 / 346

页数：8

共 58 条

[1] Human zinc fingers as building blocks in the construction of artificial transcription factors [J].

Bae, KH ;

Do Kwon, Y ;

Shin, HC ;

Hwang, MS ;

Ryu, EH ;

Park, KS ;

Yang, HY ;

Lee, D ;

Lee, Y ;

Park, J ;

Kwon, HS ;

Kim, HW ;

Yeh, BI ;

Lee, HW ;

Sohn, SH ;

Yoon, J ;

Seol, W ;

Kim, JS .

NATURE BIOTECHNOLOGY, 2003, 21 (03) :275-280

[2] CD28 costimulation and immunoaffinity-based selection efficiently generate primary gene-modified T cells for adoptive immunotherapy [J].

Berger, C ;

Blau, CA ;

Clackson, T ;

Riddell, SR ;

Heimfeld, S .

BLOOD, 2003, 101 (02) :476-484

[3] Stimulation of homologous recombination through targeted cleavage by chimeric nucleases [J].

Bibikova, M ;

Carroll, D ;

Segal, DJ ;

Trautman, JK ;

Smith, J ;

Kim, YG ;

Chandrasegaran, S .

MOLECULAR AND CELLULAR BIOLOGY, 2001, 21 (01) :289-297

[4] Enhancing gene targeting with designed zinc finger nucleases [J].

Bibikova, M ;

Beumer, K ;

Trautman, JK ;

Carroll, D .

SCIENCE, 2003, 300 (5620) :764-764

[5] Gene targeting in mice: functional analysis of the mammalian genome for the twenty-first century [J].

Capecchi, MR .

NATURE REVIEWS GENETICS, 2005, 6 (06) :507-512

[6] Progress and prospects: Zinc-finger nucleases as gene therapy agents [J].

Carroll, D. .

GENE THERAPY, 2008, 15 (22) :1463-1468

[7] Design, construction and in vitro testing of zinc finger nucleases [J].

Carroll, Dana ;

Morton, J. Jason ;

Beumer, Kelly J. ;

Segal, David J. .

NATURE PROTOCOLS, 2006, 1 (03) :1329-1341

[8] Zinc positive: tailored genome engineering meets reprogramming [J].

Cathomen, T. ;

Schambach, A. .

GENE THERAPY, 2010, 17 (01) :1-3

[9]

Cathomen Toni, 2008, V434, P277, DOI 10.1007/978-1-60327-248-3_17

[10] Zinc-finger nucleases: The next generation emerges [J].

Cathomen, Toni ;

Joung, J. Keith .

MOLECULAR THERAPY, 2008, 16 (07) :1200-1207

← 1 2 3 4 5 6 →