Gene knockout and knockin by zinc-finger nucleases: current status and perspectives

被引：26

作者：

Hauschild-Quintern, J. ^{[1
]}

Petersen, B. ^{[1
]}

Cost, G. J. ^{[3
]}

Niemann, H. ^{[1
,2
]}

机构：

[1] Friedrich Loeffler Inst, Inst Farm Anim Genet, D-31535 Neustadt, Germany

[2] Hannover Med Sch, Cluster Excellence, Hannover, Germany

[3] Sangamo BioSci, Richmond, CA 94804 USA

来源：

CELLULAR AND MOLECULAR LIFE SCIENCES | 2013年 / 70卷 / 16期

关键词：

Zinc-finger nucleases; Homology-directed repair; Transgenic animals; Gene knockout; Targeting efficiency; HOMOLOGOUS RECOMBINATION; RESTRICTION ENZYMES; MAMMALIAN-CELLS; DNA-RECOGNITION; MOUSE MODEL; GENERATION; CLEAVAGE; DISRUPTION; RATS; MUTAGENESIS;

D O I：

10.1007/s00018-012-1204-1

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Zinc-finger nucleases (ZFNs) are engineered site-specific DNA cleavage enzymes that may be designed to recognize long target sites and thus cut DNA with high specificity. ZFNs mediate permanent and targeted genetic alteration via induction of a double-strand break at a specific genomic site. Compared to conventional homology-based gene targeting, ZFNs can increase the targeting rate by up to 100,000-fold; gene disruption via mutagenic DNA repair is similarly efficient. The utility of ZFNs has been shown in many organisms, including insects, amphibians, plants, nematodes, and several mammals, including humans. This broad range of tractable species renders ZFNs a useful tool for improving the understanding of complex physiological systems, to produce transgenic animals, cell lines, and plants, and to treat human disease.

引用

页码：2969 / 2983

页数：15

共 67 条

[31] Targeted gene inactivation in zebrafish using engineered zinc-finger nucleases [J].

Meng, Xiangdong ;

Noyes, Marcus B. ;

Zhu, Lihua J. ;

Lawson, Nathan D. ;

Wolfe, Scot A. .

NATURE BIOTECHNOLOGY, 2008, 26 (06) :695-701

[32] Gene targeting by homologous recombination in mouse zygotes mediated by zinc-finger nucleases [J].

Meyer, Melanie ;

de Angelis, Martin Hrabe ;

Wurst, Wolfgang ;

Kuehn, Ralf .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2010, 107 (34) :15022-15026

[33] MPTP-induced parkinsonism in minipigs: A behavioral, biochemical, and histological study [J].

Mikkelsen, M ;

Moller, A ;

Jensen, LH ;

Pedersen, A ;

Harajehi, JB ;

Pakkenberg, H .

NEUROTOXICOLOGY AND TERATOLOGY, 1999, 21 (02) :169-175

[34] REPETITIVE ZINC-BINDING DOMAINS IN THE PROTEIN TRANSCRIPTION FACTOR IIIA FROM XENOPUS OOCYTES [J].

MILLER, J ;

MCLACHLAN, AD ;

KLUG, A .

EMBO JOURNAL, 1985, 4 (06) :1609-1614

[35] An improved zinc-finger nuclease architecture for highly specific genome editing [J].

Miller, Jeffrey C. ;

Holmes, Michael C. ;

Wang, Jianbin ;

Guschin, Dmitry Y. ;

Lee, Ya-Li ;

Rupniewski, Igor ;

Beausejour, Christian M. ;

Waite, Adam J. ;

Wang, Nathaniel S. ;

Kim, Kenneth A. ;

Gregory, Philip D. ;

Pabo, Carl O. ;

Rebar, Edward J. .

NATURE BIOTECHNOLOGY, 2007, 25 (07) :778-785

[36] Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases [J].

Moehle, E. A. ;

Rock, J. M. ;

Lee, Y. L. ;

Jouvenot, Y. ;

DeKelver, R. C. ;

Gregory, P. D. ;

Urnov, F. D. ;

Holmes, M. C. .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2007, 104 (09) :3055-3060

[37] Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology [J].

Orlando, Salvatore J. ;

Santiago, Yolanda ;

DeKelver, Russell C. ;

Freyvert, Yevgeniy ;

Boydston, Elizabeth A. ;

Moehle, Erica A. ;

Choi, Vivian M. ;

Gopalan, Sunita M. ;

Lou, Jacqueline F. ;

Li, James ;

Miller, Jeffrey C. ;

Holmes, Michael C. ;

Gregory, Philip D. ;

Urnov, Fyodor D. ;

Cost, Gregory J. .

NUCLEIC ACIDS RESEARCH, 2010, 38 (15) :e152-e152

[38] Design and selection of novel Cys2His2 zinc finger proteins [J].

Pabo, CO ;

Peisach, E ;

Grant, RA .

ANNUAL REVIEW OF BIOCHEMISTRY, 2001, 70 :313-340

[39] ZINC FINGER DNA RECOGNITION - CRYSTAL-STRUCTURE OF A ZIF268-DNA COMPLEX AT 2.1-A [J].

PAVLETICH, NP ;

PABO, CO .

SCIENCE, 1991, 252 (5007) :809-817

[40] Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases [J].

Perez, Elena E. ;

Wang, Jianbin ;

Miller, Jeffrey C. ;

Jouvenot, Yann ;

Kim, Kenneth A. ;

Liu, Olga ;

Wang, Nathaniel ;

Lee, Gary ;

Bartsevich, Victor V. ;

Lee, Ya-Li ;

Guschin, Dmitry Y. ;

Rupniewski, Igor ;

Waite, Adam J. ;

Carpenito, Carmine ;

Carroll, Richard G. ;

Orange, Jordan S. ;

Urnov, Fyodor D. ;

Rebar, Edward J. ;

Ando, Dale ;

Gregory, Philip D. ;

Riley, James L. ;

Holmes, Michael C. ;

June, Carl H. .

NATURE BIOTECHNOLOGY, 2008, 26 (07) :808-816

← 1 2 3 4 5 6 7 →