Unraveling the potential of CRISPR-Cas9 for gene therapy

被引：16

作者：

Barrangou, Rodolphe ^{[1
]}

May, Andrew P. ^{[2
]}

机构：

[1] N Carolina State Univ, Dept Food Bioproc & Nutr Sci, Raleigh, NC 27695 USA

[2] Caribou Biosci Inc, Berkeley, CA 94710 USA

来源：

EXPERT OPINION ON BIOLOGICAL THERAPY | 2015年 / 15卷 / 03期

关键词：

Cas9; CRISPR; gene therapy; genome editing; INFECTION; ENDONUCLEASE; RESISTANCE; MUTATION; PROVIDES; LATENT; MICE; DNA;

D O I：

10.1517/14712598.2015.994501

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

The molecular machinery from the prokaryotic clustered regularly interspaced short palindromic repeats (CRISPR)-Cas immune system has broadly been repurposed for genome editing in eukaryotes. In particular, the sequence-specific Cas9 endonuclease can be flexibly harnessed for the genesis of precise double-stranded DNA breaks, using single guide RNAs that are readily programmable. The endogenous DNA repair machinery subsequently generates genome modifications, either by random insertion or deletions using non-homologous end joining (NHEJ), or designed integration of mutations or genetic material using homology-directed repair (HDR) templates. This technology has opened new avenues for the investigation of genetic diseases in general, and for gene therapy applications in particular.

引用

页码：311 / 314

页数：4

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