CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes

被引:658
作者
Komor, Alexis C. [1 ,2 ,3 ]
Badran, Ahmed H. [1 ,2 ,3 ]
Liu, David R. [1 ,2 ,3 ]
机构
[1] Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
[2] Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA
[3] Broad Inst MIT & Harvard, Cambridge, MA 02141 USA
基金
美国国家科学基金会;
关键词
PLURIPOTENT STEM-CELLS; ZINC-FINGER NUCLEASES; DOUBLE-STRAND BREAKS; OFF-TARGET CLEAVAGE; DNA-CLEAVAGE; HOMOLOGOUS RECOMBINATION; KNOCK-IN; GENE DELIVERY; CAS9; PROTEIN; MOUSE MODEL;
D O I
10.1016/j.cell.2016.10.044
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
The CRISPR-Cas9 RNA-guided DNA endonuclease has contributed to an explosion of advances in the life sciences that have grown from the ability to edit genomes within living cells. In this Review, we summarize CRISPR-based technologies that enable mammalian genome editing and their various applications. We describe recent developments that extend the generality, DNA specificity, product selectivity, and fundamental capabilities of natural CRISPR systems, and we highlight some of the remarkable advancements in basic research, biotechnology, and therapeutics science that these developments have facilitated.
引用
收藏
页码:20 / 36
页数:17
相关论文
共 228 条
[91]   Systematic determination of the packaging limit of lentiviral vectors [J].
Kumar, M ;
Keller, B ;
Makalou, N ;
Sutton, RE .
HUMAN GENE THERAPY, 2001, 12 (15) :1893-1905
[92]   Genome-wide analysis reveals characteristics of off-target sites bound by the Cas9 endonuclease [J].
Kuscu, Cem ;
Arslan, Sevki ;
Singh, Ritambhara ;
Thorpe, Jeremy ;
Adli, Mazhar .
NATURE BIOTECHNOLOGY, 2014, 32 (07) :677-+
[93]   Evaluation of sgRNA Target Sites for CRISPR-Mediated Repression of TP53 [J].
Lawhorn, Ingrid E. B. ;
Ferreira, Joshua P. ;
Wang, Clifford L. .
PLOS ONE, 2014, 9 (11)
[94]   Permanent target for optical payload performance and data quality assessment: spectral characterization and a case study for calibration [J].
Li, Chuanrong ;
Ma, Lingling ;
Gao, Caixia ;
Tang, Lingli ;
Wang, Ning ;
Liu, Yaokai ;
Zhao, Yongguang ;
Dou, Shuai ;
Zhang, Dandan ;
Li, Xiaohui .
JOURNAL OF APPLIED REMOTE SENSING, 2015, 8
[95]   Heritable gene targeting in the mouse and rat using a CRISPR-Cas system [J].
Li, Dali ;
Qiu, Zhongwei ;
Shao, Yanjiao ;
Chen, Yuting ;
Guan, Yuting ;
Liu, Meizhen ;
Li, Yongmei ;
Gao, Na ;
Wang, Liren ;
Lu, Xiaoling ;
Zhao, Yongxiang ;
Liu, Mingyao .
NATURE BIOTECHNOLOGY, 2013, 31 (08) :681-683
[96]   Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9 [J].
Li, Hongmei Lisa ;
Fujimoto, Naoko ;
Sasakawa, Noriko ;
Shirai, Saya ;
Ohkame, Tokiko ;
Sakuma, Tetsushi ;
Tanaka, Michihiro ;
Amano, Naoki ;
Watanabe, Akira ;
Sakurai, Hidetoshi ;
Yamamoto, Takashi ;
Yamanaka, Shinya ;
Hotta, Akitsu .
STEM CELL REPORTS, 2015, 4 (01) :143-154
[97]   Modularly assembled designer TAL effector nucleases for targeted gene knockout and gene replacement in eukaryotes [J].
Li, Ting ;
Huang, Sheng ;
Zhao, Xuefeng ;
Wright, David A. ;
Carpenter, Susan ;
Spalding, Martin H. ;
Weeks, Donald P. ;
Yang, Bing .
NUCLEIC ACIDS RESEARCH, 2011, 39 (14) :6315-6325
[98]   TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain [J].
Li, Ting ;
Huang, Sheng ;
Jiang, Wen Zhi ;
Wright, David ;
Spalding, Martin H. ;
Weeks, Donald P. ;
Yang, Bing .
NUCLEIC ACIDS RESEARCH, 2011, 39 (01) :359-372
[99]   Simultaneous generation and germline transmission of multiple gene mutations in rat using CRISPR-Cas systems [J].
Li, Wei ;
Teng, Fei ;
Li, Tianda ;
Zhou, Qi .
NATURE BIOTECHNOLOGY, 2013, 31 (08) :684-686
[100]   Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection [J].
Liang, Xiquan ;
Potter, Jason ;
Kumar, Shantanu ;
Zou, Yanfei ;
Quintanilla, Rene ;
Sridharan, Mahalakshmi ;
Carte, Jason ;
Chen, Wen ;
Roark, Natasha ;
Ranganathan, Sridhar ;
Ravinder, Namritha ;
Chesnut, Jonathan D. .
JOURNAL OF BIOTECHNOLOGY, 2015, 208 :44-53