Mohammad Faysal Al Mazid, Olha Shkel, Eunteg Ryu, Jiwon Kim, Kyung Ho Shin, Yun Kyung Kim, Hyun Suk Lim* and Jun-Seok Lee*,
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引用次数: 0
摘要
靶蛋白降解(Target protein degradation, TPD)是通过多种细胞蛋白水解途径催化下调靶蛋白的一种很有前景的策略。尽管有许多关于新型TPD机制的报道,但发现靶向性配体仍然是一个重大挑战。与小分子配体不同,适体具有明显的优势,这是由于其基于selex的系统筛选方法。为了充分利用适体进行TPD,我们设计了一个适体和N-degron集成系统(AptaGron),该系统绕过了适体和蛋白水解招募单元之间的合成偶联。在我们的AptaGron系统中,设计了一个包含N-degron肽和与适体互补的序列的肽核酸。利用该系统,我们成功地降解了三种缺乏特异性小分子配体的靶蛋白:tau蛋白、核蛋白和真核起始因子4E (eIF4E)。我们的研究结果突出了AptaGron方法作为靶向蛋白质降解的强大平台的潜力。
Aptamer and N-Degron Ensemble (AptaGron) as a Target Protein Degradation Strategy
Target protein degradation (TPD) is a promising strategy for catalytic downregulation of target proteins through various cellular proteolytic pathways. Despite numerous reports on novel TPD mechanisms, the discovery of target-specific ligands remains a major challenge. Unlike small-molecule ligands, aptamers offer significant advantages, owing to their SELEX-based systematic screening method. To fully utilize aptamers for TPD, we designed an aptamer and N-degron ensemble system (AptaGron) that circumvents the need for synthetic conjugations between aptamers and proteolysis-recruiting units. In our AptaGron system, a peptide nucleic acid containing an N-degron peptide and a sequence complementary to the aptamer was designed. Using this system, we successfully degraded three target proteins, tau, nucleolin, and eukaryotic initiation factor 4E (eIF4E), which lack specific small-molecule ligands. Our results highlight the potential of the AptaGron approach as a robust platform for targeted protein degradation.
期刊介绍:
ACS Chemical Biology provides an international forum for the rapid communication of research that broadly embraces the interface between chemistry and biology.
The journal also serves as a forum to facilitate the communication between biologists and chemists that will translate into new research opportunities and discoveries. Results will be published in which molecular reasoning has been used to probe questions through in vitro investigations, cell biological methods, or organismic studies.
We welcome mechanistic studies on proteins, nucleic acids, sugars, lipids, and nonbiological polymers. The journal serves a large scientific community, exploring cellular function from both chemical and biological perspectives. It is understood that submitted work is based upon original results and has not been published previously.