Barrangou Rodolphe, May Andrew P
North Carolina State University, Department of Food, Bioprocessing and Nutrition Sciences , Raleigh, NC 27695 , USA +1 919 513 1644 ;
Expert Opin Biol Ther. 2015 Mar;15(3):311-4. doi: 10.1517/14712598.2015.994501. Epub 2014 Dec 23.
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.
原核生物成簇规律间隔短回文重复序列（CRISPR）-Cas免疫系统的分子机制已广泛应用于真核生物的基因组编辑。特别是，序列特异性的Cas9核酸内切酶可通过易于编程的单向导RNA灵活用于产生精确的双链DNA断裂。随后，内源性DNA修复机制通过非同源末端连接（NHEJ）随机插入或缺失，或使用同源定向修复（HDR）模板设计整合突变或遗传物质，从而产生基因组修饰。这项技术总体上为遗传疾病的研究开辟了新途径，尤其是在基因治疗应用方面。
