College of Pharmacy, Gachon University, 191 Hambakmoe-ro, Yeonsu-gu, Incheon 21936, Republic of Korea.
Gachon Pharmaceutical Research Institute, Gachon University, 191 Hambakmoe-ro, Yeonsu-gu, Incheon 21936, Republic of Korea.
Int J Mol Sci. 2023 Mar 8;24(6):5190. doi: 10.3390/ijms24065190.
Targeted protein degradation (TPD) is a promising therapeutic modality that has garnered attention in academic, industrial, and pharmaceutical research for treating diseases such as cancer, neurodegenerative disorders, inflammation, and viral infections. In this context, proteolysis-targeting chimeras (PROTACs) present a reliable technology for degrading disease-causing proteins. PROTACs complement small-molecule inhibitors, which primarily rely on direct protein regulation. From concept-to-clinic, PROTACs have evolved from cell impermeable peptide molecules to orally bioavailable drugs. Despite their potential in medicinal chemistry, certain aspects regarding PROTACs remain unclear. The clinical significance of PROTACs is primarily limited owing to their lack of selectivity and drug-like properties. This review focused on recently reported PROTAC strategies, particularly in 2022. It aimed to address and overcome the challenges posed by classical PROTACs by correlating them with emerging approaches with improved selectivity and controllability, cell permeability, linker flexibility, druggability, and PROTAC-based approaches, developed in 2022. Furthermore, recently reported PROTAC-based approaches are discussed, highlighting each of their advantages and limitations. We predict that several improved PROTAC molecules will be accessible for treating patients exhibiting various conditions, including cancer, neurodegenerative disorders, inflammation, and viral infections.
靶向蛋白降解(TPD)是一种很有前途的治疗模式,在学术、工业和制药研究中受到关注,可用于治疗癌症、神经退行性疾病、炎症和病毒感染等疾病。在这种情况下,蛋白水解靶向嵌合体(PROTAC)为降解致病蛋白提供了一种可靠的技术。PROTAC 补充了主要依赖于直接蛋白质调节的小分子抑制剂。从概念到临床,PROTAC 已经从细胞不可渗透的肽分子发展为可口服的药物。尽管它们在药物化学方面具有潜力,但 PROTAC 仍存在一些尚未明确的方面。PROTAC 的临床意义主要受到缺乏选择性和类药性的限制。本综述重点介绍了 2022 年报道的最近的 PROTAC 策略。它旨在通过将其与新兴的方法相关联,来解决和克服经典 PROTAC 面临的挑战,这些方法具有改进的选择性和可控性、细胞通透性、连接子灵活性、成药性和基于 PROTAC 的方法。此外,还讨论了最近报道的基于 PROTAC 的方法,突出了它们各自的优点和局限性。我们预计,将有几种改进的 PROTAC 分子可用于治疗表现出各种病症的患者,包括癌症、神经退行性疾病、炎症和病毒感染。