Tian Songhai, Qin Yuhang, Wu Yuxuan, Dong Min
State Key Laboratory of Natural and Biomimetic Drugs, Department of Molecular and Cellular Pharmacology, School of Pharmaceutical Sciences, Peking University, Beijing, China.
Department of Urology, Boston Children's Hospital, Boston, MA, USA.
Nat Protoc. 2025 May;20(5):1158-1195. doi: 10.1038/s41596-024-01075-y. Epub 2024 Nov 1.
Unbiased forward genetic screens have been extensively employed in biological research to elucidate functional genomics. In pooled clustered regularly interspaced short palindromic repeats (CRISPR) perturbation screens, various genetically encoded gain-of-function or loss-of-function mutations are introduced into a heterogeneous population of cells. Subsequently, these cells are screened for phenotypes, perturbation-associated genotypes are analyzed and a connection between genotype and phenotype is determined. CRISPR screening techniques enable the investigation of important biological questions, such as how bacterial toxins kill cells and cause disease. However, the broad spectrum of effects caused by diverse toxins presents a challenge when selecting appropriate screening strategies. Here, we provide a step-by-step protocol for a genome-wide pooled CRISPR perturbation screen to study bacterial toxins. We describe technical considerations, pilot experiments, library construction, screen execution, result analysis and validation of the top enriched hits. These screens are applicable for many different types of toxins and are anticipated to reveal a repertoire of host factors crucial in the intoxication pathway, such as receptors, trafficking/translocation factors and substrates. The entire protocol takes 21-27 weeks and does not require specialized knowledge beyond basic biology.
无偏正向遗传学筛选已广泛应用于生物学研究以阐明功能基因组学。在汇集的成簇规律间隔短回文重复序列(CRISPR)干扰筛选中,各种基因编码的功能获得或功能丧失突变被引入异质细胞群体中。随后,对这些细胞进行表型筛选,分析与干扰相关的基因型,并确定基因型与表型之间的联系。CRISPR筛选技术能够研究重要的生物学问题,例如细菌毒素如何杀死细胞并引发疾病。然而,在选择合适的筛选策略时,多种毒素引起的广泛效应带来了挑战。在此,我们提供了一个用于全基因组汇集CRISPR干扰筛选以研究细菌毒素的分步方案。我们描述了技术考量、预实验、文库构建、筛选执行、结果分析以及对富集程度最高的命中基因的验证。这些筛选适用于许多不同类型的毒素,预计将揭示中毒途径中至关重要的宿主因子库,如受体、运输/转运因子和底物。整个方案需要21至27周,除了基础生物学知识外不需要专门知识。