Genome-Editing Technologies: Principles and Applications

被引：190

作者：

Gaj, Thomas ^{[1
]}

Sirk, Shannon J. ^{[2
]}

Shui, Sai-lan ^{[3
]}

Liu, Jia ^{[3
]}

机构：

[1] Univ Calif Berkeley, Dept Bioengn, Berkeley, CA 94720 USA

[2] Stanford Univ, Dept Chem Engn, Stanford, CA 94305 USA

[3] ShanghaiTech Univ, Shanghai Inst Adv Immunochem Studies, Shanghai, Peoples R China

来源：

COLD SPRING HARBOR PERSPECTIVES IN BIOLOGY | 2016年 / 8卷 / 12期

基金：

美国国家卫生研究院;

关键词：

ZINC-FINGER NUCLEASES; CONTROLLING GENE-EXPRESSION; HOMOLOGY-DIRECTED REPAIR; DOUBLE-STRAND BREAKS; WIDE CRISPR SCREEN; HUMAN-CELLS; DNA-RECOGNITION; TRANSCRIPTION FACTORS; CRYSTAL-STRUCTURE; TAL EFFECTORS;

D O I：

10.1101/cshperspect.a023754

中图分类号：

Q2 [细胞生物学];

学科分类号：

071009 ; 090102 ;

摘要：

Targeted nucleases have provided researchers with the ability to manipulate virtually any genomic sequence, enabling the facile creation of isogenic cell lines and animal models for the study of human disease, and promoting exciting new possibilities for human gene therapy. Here we review three foundational technologies-clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription activator-like effector nucleases (TALENs), and zinc-finger nucleases (ZFNs). We discuss the engineering advances that facilitated their development and highlight several achievements in genome engineering that were made possible by these tools. We also consider artificial transcription factors, illustrating how this technology can complement targeted nucleases for synthetic biology and gene therapy.

引用

页数：20

共 256 条

[1] Safeguarding gene drive experiments in the laboratory [J].

Akbari, Omar S. ;

Bellen, Hugo J. ;

Bier, Ethan ;

Bullock, Simon L. ;

Burt, Austin ;

Church, George M. ;

Cook, Kevin R. ;

Duchek, Peter ;

Edwards, Owain R. ;

Esvelt, Kevin M. ;

Gantz, Valentino M. ;

Golic, Kent G. ;

Gratz, Scott J. ;

Harrison, Melissa M. ;

Hayes, Keith R. ;

James, Anthony A. ;

Kaufman, Thomas C. ;

Knoblich, Juergen ;

Malik, Harmit S. ;

Matthews, Kathy A. ;

O'Connor-Giles, Kate M. ;

Parks, Annette L. ;

Perrimon, Norbert ;

Port, Fillip ;

Russell, Steven ;

Ueda, Ryu ;

Wildonger, Jill .

SCIENCE, 2015, 349 (6251) :927-929

[2] Chimeric recombinases with designed DNA sequence recognition [J].

Akopian, A ;

He, JY ;

Boocock, MR ;

Stark, WM .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2003, 100 (15) :8688-8691

[3] Structural basis of PAM-dependent target DNA recognition by the Cas9 endonuclease [J].

Anders, Carolin ;

Niewoehner, Ole ;

Duerst, Alessia ;

Jinek, Martin .

NATURE, 2014, 513 (7519) :569-+

[4] Robust ZFN-mediated genome editing in adult hemophilic mice [J].

Anguela, Xavier M. ;

Sharma, Rajiv ;

Doyon, Yannick ;

Miller, Jeffrey C. ;

Li, Hojun ;

Haurigot, Virginia ;

Rohde, Michelle E. ;

Wong, Sunnie Y. ;

Davidson, Robert J. ;

Zhou, Shangzhen ;

Gregory, Philip D. ;

Holmes, Michael C. ;

High, Katherine A. .

BLOOD, 2013, 122 (19) :3283-3287

[5] 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

[6] Conditionally Stabilized dCas9 Activator for Controlling Gene Expression in Human Cell Reprogramming and Differentiation [J].

Balboa, Diego ;

Weltner, Jere ;

Eurola, Solja ;

Trokovic, Ras ;

Wartiovaara, Kirmo ;

Otonkoski, Timo .

STEM CELL REPORTS, 2015, 5 (03) :448-459

[7] Enabling plant synthetic biology through genome engineering [J].

Baltes, Nicholas J. ;

Voytas, Daniel F. .

TRENDS IN BIOTECHNOLOGY, 2015, 33 (02) :120-131

[8] Chemically regulated zinc finger transcription factors [J].

Beerli, RR ;

Schopfer, U ;

Dreier, B ;

Barbas, CF .

JOURNAL OF BIOLOGICAL CHEMISTRY, 2000, 275 (42) :32617-32627

[9] Toward controlling gene expression at will:: Specific regulation of the erbB-2/HER-2 promoter by using polydactyl zinc finger proteins constructed from modular building blocks [J].

Beerli, RR ;

Segal, DJ ;

Dreier, B ;

Barbas, CF .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1998, 95 (25) :14628-14633

[10] Positive and negative regulation of endogenous genes by designed transcription factors [J].

Beerli, RR ;

Dreier, B ;

Barbas, CF .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2000, 97 (04) :1495-1500

← 1 2 3 4 5 6 7 8 9 10 →