Therapeutic genome engineering via CRISPR-Cas systems

被引:21
|
作者
Moreno, Ana M. [1 ]
Mali, Prashant [1 ]
机构
[1] Univ Calif San Diego, Dept Bioengn, San Diego, CA 92103 USA
关键词
LENTIVIRAL VECTORS; HOMOLOGOUS RECOMBINATION; CELLULAR-IMMUNITY; ANALYSIS REVEALS; GENE-EXPRESSION; MOUSE MODEL; HUMAN-CELLS; RNA; DNA; EFFICIENT;
D O I
10.1002/wsbm.1380
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
Differences in genomes underlie most organismal diversity, and aberrations in genomes underlie many disease states. With the growing knowledge of the genetic and pathogenic basis of human disease, development of safe and efficient platforms for genome and epigenome engineering will transform our ability to therapeutically target human diseases and also potentially engineer disease resistance. In this regard, the recent advent of clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated (Cas) RNA-guided nuclease systems have transformed our ability to target nucleic acids. Here we review therapeutic genome engineering applications with a specific focus on the CRISPR-Cas toolsets. We summarize past and current work, and also outline key challenges and future directions. (C) 2017 Wiley Periodicals, Inc.
引用
收藏
页数:14
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