Wang Xuelian, Huang Xiumin, Fang Xiuli, Zhang Youzhong, Wang Wanpeng
Department of Gynecology and Obstetrics, Zhongshan Hospital of Xiamen University, Xiamen, China.
Department of Gynecology and Obstetrics, Qilu Hospital of Shandong University, Jinan, China.
Mol Ther Nucleic Acids. 2016 Nov 15;5(11):e388. doi: 10.1038/mtna.2016.95.
Targeted nucleases are influential instruments for intervening in genome revision with great accuracy. RNA-guided Cas9 nucleases produced from clustered regularly interspaced short palindromic repeats (CRISPR)-Cas systems have noticeably altered the means to modify the genomes of distinct organisms. They can be notably used to facilitate effective genome manipulation in eukaryotic cells by clearly detailing a 20-nt targeting sequence inside its directed RNA. We discuss the most recent advancements in the molecular basis of the type II CRISPR/Cas system and encapsulate applications and elements affecting its use in human cells. We also propose possible applications covering its uses ranging from basic science to implementation in the clinic.
靶向核酸酶是用于精确干预基因组编辑的重要工具。成簇规律间隔短回文重复序列(CRISPR)-Cas系统产生的RNA引导的Cas9核酸酶显著改变了修饰不同生物体基因组的方法。通过在其导向RNA中明确详细描述一个20个核苷酸的靶向序列,它们尤其可用于促进真核细胞中的有效基因组操作。我们讨论了II型CRISPR/Cas系统分子基础的最新进展,并概括了影响其在人类细胞中应用的应用和因素。我们还提出了可能的应用,涵盖其从基础科学到临床应用的用途。