Ward Carl C, Kleinman Jordan I, Nomura Daniel K
Departments of Chemistry, Molecular and Cell Biology, and Nutritional Sciences and Toxicology, 127 Morgan Hall, University of California, Berkeley , Berkeley, California 94720, United States.
ACS Chem Biol. 2017 Jun 16;12(6):1478-1483. doi: 10.1021/acschembio.7b00125. Epub 2017 May 1.
Most of the proteome is considered undruggable, oftentimes hindering translational efforts for drug discovery. Identifying previously unknown druggable hotspots in proteins would enable strategies for pharmacologically interrogating these sites with small molecules. Activity-based protein profiling (ABPP) has arisen as a powerful chemoproteomic strategy that uses reactivity-based chemical probes to map reactive, functional, and ligandable hotspots in complex proteomes, which has enabled inhibitor discovery against various therapeutic protein targets. Here, we report an alkyne-functionalized N-hydroxysuccinimide-ester (NHS-ester) as a versatile reactivity-based probe for mapping the reactivity of a wide range of nucleophilic ligandable hotspots, including lysines, serines, threonines, and tyrosines, encompassing active sites, allosteric sites, post-translational modification sites, protein interaction sites, and previously uncharacterized potential binding sites. Surprisingly, we also show that fragment-based NHS-ester ligands can be made to confer selectivity for specific lysine hotspots on specific targets including Dpyd, Aldh2, and Gstt1. We thus put forth NHS-esters as promising reactivity-based probes and chemical scaffolds for covalent ligand discovery.
蛋白质组的大部分被认为是不可成药的,这常常阻碍药物发现的转化研究。识别蛋白质中以前未知的可成药热点将为用小分子对这些位点进行药理学研究提供策略。基于活性的蛋白质谱分析(ABPP)已成为一种强大的化学蛋白质组学策略,它使用基于反应性的化学探针来绘制复杂蛋白质组中的反应性、功能性和可配体热点,这使得针对各种治疗性蛋白质靶点的抑制剂发现成为可能。在此,我们报道了一种炔烃功能化的N-羟基琥珀酰亚胺酯(NHS-酯),作为一种通用的基于反应性的探针,用于绘制广泛的亲核可配体热点的反应性,包括赖氨酸、丝氨酸、苏氨酸和酪氨酸,涵盖活性位点、别构位点、翻译后修饰位点、蛋白质相互作用位点以及以前未表征的潜在结合位点。令人惊讶的是,我们还表明,可以制备基于片段的NHS-酯配体,以赋予对包括Dpyd、Aldh2和Gstt1在内的特定靶点上特定赖氨酸热点的选择性。因此,我们提出NHS-酯作为有前途的基于反应性的探针和用于共价配体发现的化学支架。