Zhou Ming-Ming, Cole Philip A
Departments of Pharmacological Sciences and Oncological Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Division of Genetics, Department of Medicine, Brigham and Women's Hospital, Boston, MA, USA.
Nat Rev Drug Discov. 2025 Feb;24(2):112-133. doi: 10.1038/s41573-024-01080-6. Epub 2024 Nov 21.
Lysine acetylation is a major post-translational modification in histones and other proteins that is catalysed by the 'writer' lysine acetyltransferases (KATs) and mediates interactions with bromodomains (BrDs) and other 'reader' proteins. KATs and BrDs play key roles in regulating gene expression, cell growth, chromatin structure, and epigenetics and are often dysregulated in disease states, including cancer. There have been accelerating efforts to identify potent and selective small molecules that can target individual KATs and BrDs with the goal of developing new therapeutics, and some of these agents are in clinical trials. Here, we summarize the different families of KATs and BrDs, discuss their functions and structures, and highlight key advances in the design and development of chemical agents that show promise in blocking the action of these chromatin proteins for disease treatment.
赖氨酸乙酰化是组蛋白和其他蛋白质中一种主要的翻译后修饰,由“书写者”赖氨酸乙酰转移酶(KATs)催化,并介导与溴结构域(BrDs)和其他“读取者”蛋白质的相互作用。KATs和BrDs在调节基因表达、细胞生长、染色质结构和表观遗传学方面发挥着关键作用,并且在包括癌症在内的疾病状态中常常失调。人们一直在加速努力,以鉴定能够靶向单个KATs和BrDs的强效且选择性的小分子,目的是开发新的治疗方法,其中一些药物正在进行临床试验。在此,我们总结了KATs和BrDs的不同家族,讨论了它们的功能和结构,并强调了在设计和开发化学药物方面取得的关键进展,这些药物有望阻断这些染色质蛋白的作用以用于疾病治疗。