Li Jiaying, Wang Guosheng, Ye Mingliang, Qin Hongqiang
1. College of Chemical Engineering, Shenyang University of Chemical Technology, Shenyang 110142, China;2. CAS Key Laboratory of Separation Science for Analytical Chemistry, Dalian Institute of Chemical Physics, Chinese Academy of Sciences, Dalian 116023, China.
1. College of Chemical Engineering, Shenyang University of Chemical Technology, Shenyang 110142, China.
Se Pu. 2023 Jan;41(1):14-23. doi: 10.3724/SP.J.1123.2022.05013.
The discovery of novel drug targets enhances the development of novel drugs, and the discovery of novel target proteins depends on highly accurate high-throughput methods of analyzing drug-protein interactions. Protein expression levels, spatial localization, and structural differences directly affect pharmacodynamics. To date, >20000 proteins have been discovered in the human proteome by the genome and proteome projects via gene and protein sequencing. Understanding the biological functions of proteins is critical in identifying and regulating biological processes, with most remaining unidentified. Until recently, >85% of proteins were considered undruggable, mainly because of the lack of binding pockets and active sites targeted by small molecules. Therefore, characterization of the reactive sites of amino acids based on proteomic hierarchy is the key to novel drug design. Recently, with the rapid development of mass spectrometry (MS), the study of drug-target protein interactions based on proteomics technology has been considerably promoted. Activity-based protein profiling (ABPP) is an active chemical probe-based method of detecting functional enzymes and drug targets in complex samples. Compared with classical proteomics strategies, ABPP is based mainly on protein activity. It has been successfully utilized to characterize the activities of numerous protease families with crucial biological functions, such as serine hydrolases, protein kinases, glycosidases, and metalloenzymes. It has also been used to identify key enzymes that are closely related to diseases and develop covalent inhibitors for use in disease treatment. The technology used in proteome analysis ranges from gel electrophoresis to high-throughput MS due to the progress of MS technology. ABPP strategies combined with chemical probe labeling and quantitative MS enable the characterization of amino acid activity, which may enhance the discovery of novel drug targets and the development of lead compounds. Amino acid residues play critical roles in protein structures and functions, and covalent drugs targeting these amino acids are effective in treating numerous diseases. There are 20 main types of natural amino acids, with different reactivities, in the proteins in the human body. In addition, the proteins and amino acids are affected by the spatial microenvironment, leading to significant differences in their spatial reactivities. The key in evaluating the reactivities of amino acids via ABPP is to select those with high reactivities. The core of the ABPP strategy is the use of chemical probes to label amino acid sites that exhibit higher activities in certain environments. The activity-based probe (ABP) at the core of ABPP consists of three components: reactive, reporter groups and a linker. The reactive group is the basis of the ABP and anchors the drug target via strong forces, such as covalent bonds. The reaction exhibits a high specificity and conversion rate and should display a good biocompatibility. Activity probes based on different amino acid residues have been developed, and the screening of amino acid activity combined with isotope labeling is a new focus of research. Currently, different types of ABPs have been developed to target amino acids and characterize amino acid reactivity, such as cysteine labeled with an electrophilic iodoacetamide probe and lysine labeled with activated esters. ABPP facilitates the discovery of potentially therapeutic protein targets, the screening of lead compounds, and the identification of drug targets, thus aiding the design of novel drugs. This review focuses on the development of ABPP methods and the progress in the screening of amino acid reactivity using ABPs, which should be promising methods for use in designing targeted drugs with covalent interactions.
新型药物靶点的发现促进了新型药物的研发,而新型靶点蛋白的发现依赖于分析药物 - 蛋白质相互作用的高精度高通量方法。蛋白质表达水平、空间定位和结构差异直接影响药效学。迄今为止,基因组和蛋白质组计划通过基因和蛋白质测序在人类蛋白质组中发现了超过20000种蛋白质。了解蛋白质的生物学功能对于识别和调节生物过程至关重要,但大多数蛋白质的功能仍未明确。直到最近,超过85%的蛋白质被认为是不可成药的,主要是因为缺乏小分子靶向的结合口袋和活性位点。因此,基于蛋白质组层次结构对氨基酸反应位点进行表征是新型药物设计的关键。近年来,随着质谱(MS)技术的快速发展,基于蛋白质组学技术的药物 - 靶点蛋白相互作用研究得到了显著推动。基于活性的蛋白质谱分析(ABPP)是一种基于活性化学探针的方法,用于检测复杂样品中的功能酶和药物靶点。与经典蛋白质组学策略相比,ABPP主要基于蛋白质活性。它已成功用于表征众多具有关键生物学功能的蛋白酶家族的活性,如丝氨酸水解酶、蛋白激酶、糖苷酶和金属酶。它还被用于鉴定与疾病密切相关的关键酶,并开发用于疾病治疗的共价抑制剂。由于MS技术的进步,蛋白质组分析中使用的技术范围从凝胶电泳到高通量MS。ABPP策略结合化学探针标记和定量MS能够表征氨基酸活性,这可能会促进新型药物靶点的发现和先导化合物的开发。氨基酸残基在蛋白质结构和功能中起着关键作用,靶向这些氨基酸的共价药物在治疗多种疾病方面有效。人体蛋白质中存在20种主要类型的天然氨基酸,它们具有不同的反应性。此外,蛋白质和氨基酸受到空间微环境的影响,导致它们的空间反应性存在显著差异。通过ABPP评估氨基酸反应性的关键是选择那些具有高反应性的氨基酸。ABPP策略的核心是使用化学探针标记在特定环境中表现出较高活性的氨基酸位点。ABPP核心的基于活性的探针(ABP)由三个部分组成:反应基团、报告基团和连接子。反应基团是ABP的基础,通过共价键等强力锚定药物靶点。该反应具有高特异性和转化率,并且应表现出良好的生物相容性。已经开发了基于不同氨基酸残基的活性探针,结合同位素标记筛选氨基酸活性是一个新的研究重点。目前,已经开发了不同类型的ABP来靶向氨基酸并表征氨基酸反应性,如用亲电碘乙酰胺探针标记的半胱氨酸和用活化酯标记的赖氨酸。ABPP有助于发现潜在的治疗性蛋白质靶点、筛选先导化合物以及鉴定药物靶点,从而有助于新型药物的设计。本综述重点介绍了ABPP方法的发展以及使用ABP筛选氨基酸反应性的进展,这应该是用于设计具有共价相互作用的靶向药物的有前景的方法。