Clustered regularly interspaced short palindromic repeat/CRISPR-associated 9 (CRISPR/Cas9) enables targeted genome engineering. The simplicity of this system, its facile engineering, and amenability to multiplex genes make it the system of choice for many applications. This system has revolutionized our ability to carry out gene editing, transcription regulation, genome imaging, and epigenetic modification. In this review, we discuss the discovery of CRISPR/Cas9, its mechanism of action, its application in medicine and animal model development, and its delivery. We also highlight how the CRISPR/Cas9 system can affect the next generation of drugs by accelerating the identification and validation of high-value targets. The generation of precision disease models through this system will provide a rapid avenue for functional drug screening.

聚簇的规则间隔的短回文重复序列/ CRISPR相关的9（CRISPR / Cas9）使得靶向基因组工程化成为可能。该系统的简单性，简便的工程设计以及对多基因的适应性使其成为许多应用的首选系统。该系统彻底改变了我们进行基因编辑，转录调控，基因组成像和表观遗传修饰的能力。在这篇综述中，我们讨论了CRISPR / Cas9的发现，其作用机理，其在医学和动物模型开发中的应用以及其递送。我们还将重点介绍CRISPR / Cas9该系统可以通过加快对高价值目标的识别和验证来影响下一代药物。通过该系统生成的精确疾病模型将为功能药物筛选提供快速途径。