使用环境敏感报告基因对protac介导的蛋白质降解进行无创体内监测

IF 15.7 1区 综合性期刊 Q1 MULTIDISCIPLINARY SCIENCES
Tao Li, Qingyu Zong, He Dong, Ihsan Ullah, Zhenhai Pan, Youyong Yuan
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引用次数: 0

摘要

靶向嵌合体蛋白水解(Proteolysis targeting chimeras, PROTACs)是一项突破性的治疗技术,用于选择性降解感兴趣的蛋白(POIs)。PROTACs的结构变化不可预测地影响了它们的蛋白质降解效率,这主要是通过western blotting定量POIs丰度来评估的。然而,这种方法无法实现对活细胞内蛋白质降解的非侵入性监测,更不用说直接评估体内的降解效果了。在此,我们开发了一种环境敏感报告基因(ESR),用于定量体内由PROTACs触发的蛋白质降解事件。通过同时整合poi靶向配体和环境敏感荧光团,ESR信号与poi水平表现出很强的荧光相关性。这种无创监测报告提供了一种高通量和方便的方法来筛选靶向降解物的POIs,并预测protacs介导的小鼠模型的治疗结果。这些特性表明,ESR策略有潜力作为一种通用的模块化方案,用于非侵入性定量癌症相关治疗靶点的蛋白质降解。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Non-invasive in vivo monitoring of PROTAC-mediated protein degradation using an environment-sensitive reporter

Non-invasive in vivo monitoring of PROTAC-mediated protein degradation using an environment-sensitive reporter

Proteolysis targeting chimeras (PROTACs) represent a groundbreaking therapeutic technology for selectively degrading proteins of interest (POIs). The structural variations in PROTACs unpredictably influence their protein degradation efficiency, which is predominantly assessed by quantifying POIs abundance through western blotting. This approach, however, falls short of enabling non-invasive monitoring of protein degradation within living cells let alone assessing directly the degradation effects in vivo. Herein, we develop an environment-sensitive reporter (ESR) for the quantification of protein degradation events triggered by PROTACs in vivo. By simultaneously integrating POIs targeting ligand and an environment-sensitive fluorophore, the ESR signals exhibit a strong fluorescence correlation with the levels of POIs. This non-invasive monitoring reporter offers a high-throughput and convenient way to screen POIs targeting degraders and predict PROTACs-mediated therapeutic outcomes in mouse models. These properties suggest the potential of ESR strategy as a general modular scheme for non-invasive quantification of protein degradation of cancer-related therapeutic targets.

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来源期刊
Nature Communications
Nature Communications Biological Science Disciplines-
CiteScore
24.90
自引率
2.40%
发文量
6928
审稿时长
3.7 months
期刊介绍: Nature Communications, an open-access journal, publishes high-quality research spanning all areas of the natural sciences. Papers featured in the journal showcase significant advances relevant to specialists in each respective field. With a 2-year impact factor of 16.6 (2022) and a median time of 8 days from submission to the first editorial decision, Nature Communications is committed to rapid dissemination of research findings. As a multidisciplinary journal, it welcomes contributions from biological, health, physical, chemical, Earth, social, mathematical, applied, and engineering sciences, aiming to highlight important breakthroughs within each domain.
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