Institute of Cell and Development Biology, College of Life Sciences, Zijingang Campus, Zhejiang University, Hangzhou, Zhejiang, P.R. China.
Institute of Orthopedics, Xiaoshan Traditional Chinese Medical Hospital, Hangzhou, Zhejiang, China.
Hum Mol Genet. 2018 Aug 1;27(R2):R79-R88. doi: 10.1093/hmg/ddy120.
Genomic editing to correct disease-causing mutations is a promising approach for the treatment of human diseases. As a simple and programmable nuclease-based genomic editing tool, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system has substantially improved the ability to make precise changes in the human genome. Rapid development of CRISPR-based technologies in recent years has expanded its application scope and promoted CRISPR-based therapies in preclinical trails. Here, we review the application of the CRISPR system over the last 2 years; including its development and application in base editing, transcription modulation and epigenetic editing, genomic-scale screening, and cell and embryo therapy. Finally, the prospects and challenges related to application of CRISPR/Cas9 technologies are discussed.
基因组编辑纠正致病突变是治疗人类疾病的一种很有前途的方法。作为一种简单且可编程的基于核酸酶的基因组编辑工具,成簇规律间隔短回文重复序列 (CRISPR)/CRISPR 相关蛋白 9 (Cas9) 系统大大提高了在人类基因组中进行精确改变的能力。近年来,基于 CRISPR 的技术的快速发展扩大了其应用范围,并促进了基于 CRISPR 的疗法进入临床前试验。在这里,我们回顾了过去 2 年中 CRISPR 系统的应用;包括其在碱基编辑、转录调控和表观遗传编辑、基因组规模筛选以及细胞和胚胎治疗中的发展和应用。最后,讨论了与 CRISPR/Cas9 技术应用相关的前景和挑战。