新一代基因组编辑系统CRISPR/Cpf1

The new generation tool for CRISPR genome editing: CRISPR/Cpf1

摘要:

CRISPR/Cas系统几乎存在于所有的细菌和古菌中，是用来抵御外来病毒和噬菌体入侵的获得性免疫防御机制。2012年起CRISPR/Cas9被改造为基因编辑工具，并衍生出一系列高效、便捷的基因编辑工具，迅速在基础理论、基因诊断和临床治疗等研究领域中得到广泛应用。然而，CRISPR/Cas9也存在细胞毒性、脱靶效应和基因插入困难等一些亟待解决的问题，在一定程度上限制了CRISPR/Cas9的应用。Cpf1是2015年报道的一种新型CRISPR效应蛋白，具有许多与Cas9不同的特性，有利于克服CRISPR/Cas9应用中的一些限制。本文综述了近两年来对CRISPR/Cpf1的研究进展和应用，并对其应用前景和发展方向进行了展望。

Abstract:

Almost all archaea and many bacteria achieve adaptive immunity through a diverse set of CRISPR-Cas systems. From 2012, Cas9 has been harnessed by thousands of laboratories for genome editing applications in a variety of experimental model systems. Cas9 is driving innovative applications from basic biology to biotechnology and medicine. However, numerous challenges still remain, such as limited targeting range, toxicity, and potential for off-target mutagenesis. Cpf1 represents a class 2/type V CRISPR RNA-guided endonuclease that is distinct from the type II CRISPR Cas9 nuclease. Future exploration of strategies using Cpf1 is expected to bring solutions for some of the biggest challenges facing genome editing. Here, we review the development and applications of Cpf1 for a variety of research or applications, and highlight challenges as well as future directions.