Larrimore Katherine E, Serebrenik Yevgeniy V
Department of Biochemistry and Molecular Biology, Robert Wood Johnson Medical School, Rutgers University, Piscataway, NJ, United States.
Front Pharmacol. 2025 Jun 23;16:1593844. doi: 10.3389/fphar.2025.1593844. eCollection 2025.
Multifunctional ligand-binding domains have revolutionized cell biology by enabling the precise visualization and manipulation of fused proteins of interest through small molecule probes. Employing these domains is labor-intensive and low-throughput, limiting studies to only small subsets of proteins at a time. However, advancements in high-throughput technologies like pooled protein tagging have enabled the tagging of proteins across the entire proteome with ligand-binding domains. This allows for the generation of complex cell libraries where each cell expresses a different protein fused to a generic, ligandable handle. These libraries unlock opportunities to explore the proteome with a versatile toolbox of small molecule probes designed to interact with the fused tags, allowing researchers to visualize protein localization, induce protein misfolding, manipulate protein-protein interactions, modulate protein stability, and more in a scalable, systematic manner. This review explores recent studies employing multifunctional domains at proteome scale, delving into how the associated chemical probes have been employed to enable insights into endogenous protein function, cellular processes, and disease mechanisms. Additional multifunctional ligand-binding domains are discussed that can be used in pooled protein tagging, as well as their potential strengths and weaknesses. We also discuss potential applications in drug discovery such as high-throughput screening for therapeutic targets with insights for bifunctional ligand optimization. By integrating pooled protein tagging with ligand-binding domains and chemical probes, we highlight how the fusion of chemical biology and functional genomics is paving the way for innovative research avenues and transformative advances in cell biology and pharmacology.
多功能配体结合结构域通过小分子探针实现对感兴趣的融合蛋白进行精确可视化和操作,从而彻底改变了细胞生物学。使用这些结构域需要耗费大量人力且通量较低,一次只能对少量蛋白质子集进行研究。然而,诸如汇集蛋白质标签等高通量技术的进步使得能够用配体结合结构域对整个蛋白质组中的蛋白质进行标记。这使得能够生成复杂的细胞文库,其中每个细胞表达与通用的、可结合配体的手柄融合的不同蛋白质。这些文库为利用设计用于与融合标签相互作用的小分子探针通用工具箱探索蛋白质组提供了机会,使研究人员能够以可扩展、系统的方式可视化蛋白质定位、诱导蛋白质错误折叠、操纵蛋白质 - 蛋白质相互作用、调节蛋白质稳定性等。本综述探讨了在蛋白质组规模上使用多功能结构域的最新研究,深入研究了如何利用相关化学探针来深入了解内源性蛋白质功能、细胞过程和疾病机制。还讨论了可用于汇集蛋白质标签的其他多功能配体结合结构域,以及它们潜在的优缺点。我们还讨论了在药物发现中的潜在应用,例如高通量筛选治疗靶点以及对双功能配体优化的见解。通过将汇集蛋白质标签与配体结合结构域和化学探针相结合,我们强调了化学生物学与功能基因组学的融合如何为细胞生物学和药理学中的创新研究途径和变革性进展铺平道路。