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靶向蛋白降解剂作为潜在治疗药物的最新进展。

Recent advances in targeted protein degraders as potential therapeutic agents.

作者信息

Yang Na, Kong Bo, Zhu Zhaohong, Huang Fei, Zhang Liliang, Lu Tao, Chen Yadong, Zhang Yanmin, Jiang Yulei

机构信息

School of Science, China Pharmaceutical University, 639 Longmian Avenue, Nanjing, 211198, People's Republic of China.

State Key Laboratory of Natural Medicines, China Pharmaceutical University, 24 Tongjiaxiang, Nanjing, 210009, People's Republic of China.

出版信息

Mol Divers. 2024 Feb;28(1):309-333. doi: 10.1007/s11030-023-10606-w. Epub 2023 Feb 15.

Abstract

Targeted protein degradation (TPD) technology has gradually become widespread in the past 20 years, which greatly boosts the development of disease treatment. Contrary to small inhibitors that act on protein kinases, transcription factors, ion channels, and other targets they can bind to, targeted protein degraders could target "undruggable targets" and overcome drug resistance through ubiquitin-proteasome pathway (UPP) and lysosome pathway. Nowadays, some bivalent degraders such as proteolysis-targeting chimeras (PROTACs) have aroused great interest in drug discovery, and some of them have successfully advanced into clinical trials. In this review, to better understand the mechanism of degraders, we elucidate the targeted protein degraders according to their action process, relying on the ubiquitin-proteasome system or lysosome pathway. Then, we briefly summarize the study of PROTACs employing different E3 ligases. Subsequently, the effect of protein of interest (POI) ligands, linker, and E3 ligands on PROTAC degradation activity is also discussed in detail. Other novel technologies based on UPP and lysosome pathway have been discussed in this paper such as in-cell click-formed proteolysis-targeting chimeras (CLIPTACs), molecular glues, Antibody-PROTACs (Ab-PROTACs), autophagy-targeting chimeras, and lysosome-targeting chimeras. Based on the introduction of these degradation technologies, we can clearly understand the action process and degradation mechanism of these approaches. From this perspective, it will be convenient to obtain the development status of these drugs, choose appropriate degradation methods to achieve better disease treatment and provide basis for future research and simultaneously distinguish the direction of future research efforts.

摘要

在过去20年里,靶向蛋白质降解(TPD)技术已逐渐广泛应用,极大地推动了疾病治疗的发展。与作用于蛋白激酶、转录因子、离子通道等可结合靶点的小分子抑制剂不同,靶向蛋白质降解剂可针对“不可成药靶点”,并通过泛素-蛋白酶体途径(UPP)和溶酶体途径克服耐药性。如今,一些双功能降解剂,如蛋白酶靶向嵌合体(PROTACs),在药物研发中引起了极大关注,其中一些已成功进入临床试验。在本综述中,为了更好地理解降解剂的作用机制,我们根据其作用过程,依据泛素-蛋白酶体系统或溶酶体途径对靶向蛋白质降解剂进行了阐释。然后,我们简要总结了采用不同E3连接酶的PROTACs的研究情况。随后,还详细讨论了目标蛋白(POI)配体、连接子和E3配体对PROTAC降解活性的影响。本文还讨论了基于UPP和溶酶体途径的其他新技术,如细胞内点击形成的蛋白酶靶向嵌合体(CLIPTACs)、分子胶、抗体-PROTACs(Ab-PROTACs)、自噬靶向嵌合体和溶酶体靶向嵌合体。基于对这些降解技术的介绍,我们可以清楚地了解这些方法的作用过程和降解机制。从这个角度来看,将便于了解这些药物的研发状况,选择合适的降解方法以实现更好的疾病治疗,并为未来研究提供依据,同时明确未来研究工作的方向。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fb2a/9930057/c5c254fbb2f0/11030_2023_10606_Fig1_HTML.jpg

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