Cancer Research Institute, School of Basic Medical Sciences, State Key Laboratory of Organ Failure Research, National Clinical Research Center of Kidney Disease, Key Laboratory of Organ Failure Research (Ministry of Education), Southern Medical University, Guangzhou, China.
Department of Nephrology, Shenzhen Hospital, Southern Medical University, Shenzhen, China.
J Gene Med. 2021 Nov;23(11):e3377. doi: 10.1002/jgm.3377. Epub 2021 Jul 28.
The clustered regularly interspaced short palindromic repeats (CRISPR) system is a state-of-the-art tool for versatile genome editing that has advanced basic research dramatically, with great potential for clinic applications. The system consists of two key molecules: a CRISPR-associated (Cas) effector nuclease and a single guide RNA. The simplicity of the system has enabled the development of a wide spectrum of derivative methods. Almost any laboratory can utilize these methods, although new users may initially be confused when faced with the potentially overwhelming abundance of choices. Cas nucleases and their engineering have been systematically reviewed previously. In the present review, we discuss single guide RNA engineering and design strategies that facilitate more efficient, more specific and safer gene editing.
成簇规律间隔短回文重复序列 (CRISPR) 系统是一种用于多功能基因组编辑的先进工具，极大地推动了基础研究的发展，具有巨大的临床应用潜力。该系统由两种关键分子组成：CRISPR 相关（Cas）效应核酸酶和单链向导 RNA。该系统的简单性使得衍生方法的种类繁多。几乎任何实验室都可以使用这些方法，尽管新用户在面对可能压倒性的大量选择时可能会感到困惑。Cas 核酸酶及其工程已被系统地综述过。在本综述中，我们讨论了单链向导 RNA 的工程和设计策略，这些策略可以促进更高效、更特异和更安全的基因编辑。
