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Cas9-Based Tools for Targeted Genome Editing and Transcriptional Control

机译：用于目标基因组编辑和转录控制的基于Cas9的工具

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Development of tools for targeted genome editing and regulation of gene expression has significantly expanded our ability to elucidate the mechanisms of interesting biological phenomena and to engineer desirable biological systems. Recent rapid progress in the study of a clustered, regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated (Cas) protein system in bacteria has facilitated the development of newly facile and programmable platforms for genome editing and transcriptional control in a sequence-specific manner. The core RNA-guided Cas9 endonuclease in the type II CRISPR system has been harnessed to realize gene mutation and DNA deletion and insertion, as well as transcriptional activation and repression, with multiplex targeting ability, just by customizing 20-nucleotide RNA components. Here we describe the molecular basis of the type II CRISPR/Cas system and summarize applications and factors affecting its utilization in model organisms. We also discuss the advantages and disadvantages of Cas9-based tools in comparison with widely used customizable tools, such as Zinc finger nucleases and transcription activator-like effector nucleases.
机译：用于靶向基因组编辑和基因表达调控的工具的开发已大大扩展了我们阐明有趣的生物学现象的机制和设计理想的生物学系统的能力。细菌中簇状，规则间隔的短回文重复（CRISPR）/ CRISPR相关（Cas）蛋白系统的研究方面的最新快速进展促进了新的灵活且可编程的平台的开发，该平台可用于以序列特异性方式进行基因组编辑和转录控制方式。仅通过定制20个核苷酸的RNA组件，即可利用II型CRISPR系统中核心的RNA引导的Cas9内切核酸酶实现基因突变和DNA缺失和插入，以及转录激活和抑制，具有多重靶向能力。在这里，我们描述了II型CRISPR / Cas系统的分子基础，并总结了其在模型生物中的应用及其影响因素。与广泛使用的可定制工具（例如锌指核酸酶和转录激活因子样效应子核酸酶）相比，我们还讨论了基于Cas9的工具的优缺点。

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