Department of Genetics, Harvard Medical School, Boston, Massachusetts, USA.
Nat Methods. 2013 Oct;10(10):957-63. doi: 10.1038/nmeth.2649.
RNA-guided Cas9 nucleases derived from clustered regularly interspaced short palindromic repeats (CRISPR)-Cas systems have dramatically transformed our ability to edit the genomes of diverse organisms. We believe tools and techniques based on Cas9, a single unifying factor capable of colocalizing RNA, DNA and protein, will grant unprecedented control over cellular organization, regulation and behavior. Here we describe the Cas9 targeting methodology, detail current and prospective engineering advances and suggest potential applications ranging from basic science to the clinic.
RNA 引导的 Cas9 核酸酶来源于成簇规律间隔短回文重复序列 (CRISPR)-Cas 系统,极大地改变了我们编辑多种生物基因组的能力。我们相信,基于 Cas9 的工具和技术——一种能够使 RNA、DNA 和蛋白质共定位的单一统一因素——将赋予对细胞组织、调控和行为前所未有的控制能力。在此,我们描述了 Cas9 的靶向方法,详细介绍了当前和未来的工程进展,并提出了从基础科学到临床应用的潜在应用。