Functional genetic screening as an important method for exploring biological processes, diseases development research and functional annotation of genetic elements, has been widely used in pharmaceutical research, new therapeutic targets identifying and screening, and tumor resistance. CRISPR-Cas9 (Clustered regularly interspaced short palindromic repeat sequences/CRISPR-associated protein 9) is the newest tool in the geneticist's toolbox, allowing researchers to edit genome with unprecedented ease, accuracy and high-throughput. CRISPR-Cas9 system provides a high-throughput, practical and efficient tool for the discovery of functionally important genes responsible for certain phenotypes. In this review, we summarize the characterization of CRISPR/Cas9 system and applications of this new genetic toolkit in functional genetic screening.
功能性基因的筛选是探索生物进程、研究疾病发生发展和诠释基因功能的重要方法，在生物、医药、新治疗靶点筛选及肿瘤耐药等方面有广泛的应用。CRISPR-Cas9 (Clustered regularly interspaced short palindromic repeat sequences/CRISPR-associated protein 9) 技术作为近期热门的基因编辑工具，能够高通量地对基因组进行精准修饰，为实现功能性基因的筛选提供了简便高效的技术支持。文中对CRISPR-cas9 技术应用于功能性基因筛选的方法及研究进展进行了综述。.
Keywords: CRISPR/Cas9; functional genetic screening; genome-scale knockout library.