Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9

被引:329
|
作者
Gupta, Rajat M.
Musunuru, Kiran
机构
[1] Harvard Univ, Dept Stem Cell & Regenerat Biol, Cambridge, MA 02138 USA
[2] Brigham & Womens Hosp, Div Cardiovasc Med, Boston, MA 02115 USA
来源
JOURNAL OF CLINICAL INVESTIGATION | 2014年 / 124卷 / 10期
关键词
ZINC-FINGER NUCLEASES; ONE-STEP GENERATION; HUMAN-CELLS; HOMOLOGOUS RECOMBINATION; EMBRYO MICROINJECTION; KNOCKOUT RATS; GENOME MODIFICATION; EFFECTOR NUCLEASES; BETA-GLOBIN; HUMAN IPSCS;
D O I
10.1172/JCI72992
中图分类号
R-3 [医学研究方法]; R3 [基础医学];
学科分类号
1001 ;
摘要
The past decade has been one of rapid innovation in genome-editing technology. The opportunity now exists for investigators to manipulate virtually any gene in a diverse range of cell types and organisms with targeted nucleases designed with sequence-specific DNA-binding domains. The rapid development of the field has allowed for highly efficient, precise, and now cost-effective means by which to generate human and animal models of disease using these technologies. This review will outline the recent development of genome-editing technology, culminating with the use of CRISPR-Cas9 to generate novel mammalian models of disease. While the road to using this same technology for treatment of human disease is long, the pace of innovation over the past five years and early successes in model systems build-anticipation for this prospect.
引用
收藏
页码:4154 / 4161
页数:8
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