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荧光偶联泛素化检测作为一种新型脑啡肽酶降解剂的高通量筛选策略

Fluorescence-Coupled Ubiquitination Assay as a High-Throughput Screening Strategy for Novel Cereblon Degraders.

作者信息

Deng Yanan, Yang Shiling, Xu Hesong, Ding Xiyao, Xu Ying, Ye Zhengzheng, Chen Yan, Zhang Zemin, Lin Jin, Xiong Huan, Zhang Zizhong, Yang Kun, Hu Yiran, Xu Ke, Luo Cheng, Chen Shijie, Lin Hua, Li Zhihai

机构信息

School of Pharmaceutical Science and Technology, Hangzhou Institute for Advanced Study, University of Chinese Academy of Sciences, Hangzhou 310024, China.

State Key Laboratory of Drug Research, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, 555 Zuchongzhi Road, Shanghai 201203, China.

出版信息

J Med Chem. 2025 May 22;68(10):10111-10127. doi: 10.1021/acs.jmedchem.5c00065. Epub 2025 May 7.

Abstract

Cereblon (CRBN)-based protein degradation, via molecular glue degraders (MGDs) and proteolysis-targeting chimeras (PROTACs), is a promising cancer treatment strategy in targeted protein degradation (TPD). However, novel degraders discovery remains limited due to the lack of robust, high-throughput screening (HTS) methods for processing pools of purified compounds or complex chemical synthesis mixtures. Here, we introduce an innovative HTS strategy that employs a highly sensitive, fluorescence-coupled ubiquitination assay to identify CRBN-based degraders. This approach tracks ubiquitinated target proteins via gel-based analyses, and thereby progressively narrows down the list of potential degrader molecules from large-scale compound libraries or chemical reaction mixtures. Using this strategy, we identified LL-BPTF-8, a promising lead compound of PROTAC degrader with high potency and selectivity that targets the bromodomain PHD finger transcription factor (BPTF). Overall, our method offers a low-cost, rapid, and versatile platform for the HTS of protein degrader candidates, significantly streamlining the discovery of novel degraders.

摘要

基于脑啡肽(CRBN)的蛋白质降解,通过分子胶降解剂(MGD)和蛋白酶靶向嵌合体(PROTAC),是靶向蛋白质降解(TPD)中一种很有前景的癌症治疗策略。然而,由于缺乏用于处理纯化化合物池或复杂化学合成混合物的强大高通量筛选(HTS)方法,新型降解剂的发现仍然有限。在此,我们介绍一种创新的HTS策略,该策略采用高度灵敏的荧光偶联泛素化测定法来鉴定基于CRBN的降解剂。这种方法通过基于凝胶的分析追踪泛素化的靶蛋白,从而逐步从大规模化合物库或化学反应混合物中缩小潜在降解剂分子的范围。使用这种策略,我们鉴定出LL-BPTF-8,这是一种有前景的PROTAC降解剂先导化合物,对靶向溴结构域PHD指转录因子(BPTF)具有高效力和选择性。总体而言,我们的方法为蛋白质降解剂候选物的HTS提供了一个低成本、快速且通用的平台,显著简化了新型降解剂的发现过程。

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