Annual Review of Biochemistry ( IF 12.1 ) Pub Date : 2016-06-13 00:00:00 , DOI: 10.1146/annurev-biochem-060815-014607 Haifeng Wang 1 , Marie La Russa 1, 2 , Lei S Qi 1, 3, 4
The Cas9 protein (CRISPR-associated protein 9), derived from type II CRISPR (clustered regularly interspaced short palindromic repeats) bacterial immune systems, is emerging as a powerful tool for engineering the genome in diverse organisms. As an RNA-guided DNA endonuclease, Cas9 can be easily programmed to target new sites by altering its guide RNA sequence, and its development as a tool has made sequence-specific gene editing several magnitudes easier. The nuclease-deactivated form of Cas9 further provides a versatile RNA-guided DNA-targeting platform for regulating and imaging the genome, as well as for rewriting the epigenetic status, all in a sequence-specific manner. With all of these advances, we have just begun to explore the possible applications of Cas9 in biomedical research and therapeutics. In this review, we describe the current models of Cas9 function and the structural and biochemical studies that support it. We focus on the applications of Cas9 for genome editing, regulation, and imaging, discuss other possible applications and some technical considerations, and highlight the many advantages that CRISPR/Cas9 technology offers.
中文翻译：
CRISPR/Cas9 在基因组编辑及其他领域的应用
Cas9 蛋白（CRISPR 相关蛋白 9）源自 II 型 CRISPR（成簇规则间隔的短回文重复序列）细菌免疫系统，正在成为多种生物体基因组工程的强大工具。作为一种 RNA 引导的 DNA 核酸内切酶，Cas9 可以通过改变其引导 RNA 序列轻松编程以靶向新位点，并且它作为工具的开发使序列特异性基因编辑变得更加容易。 Cas9 的核酸酶失活形式进一步提供了一个多功能的 RNA 引导的 DNA 靶向平台，用于调节和成像基因组，以及重写表观遗传状态，所有这些都以序列特异性的方式进行。凭借所有这些进展，我们才刚刚开始探索 Cas9 在生物医学研究和治疗中的可能应用。在这篇综述中，我们描述了 Cas9 功能的当前模型以及支持它的结构和生化研究。我们重点关注 Cas9 在基因组编辑、调控和成像方面的应用，讨论其他可能的应用和一些技术考虑因素，并强调 CRISPR/Cas9 技术提供的许多优势。
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