A Small-Molecule Drug for the Self-Checking of Mitophagy.

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Tác giả: Qingjie Bai, Qixin Chen, Xiaoyuan Chen, Yanan Gao, Tony D James, Simon E Lewis, Youxiao Ren, Xintian Shao, Luling Wu, Mengrui Zhang

Ngôn ngữ: eng

Ký hiệu phân loại: 133.594 Types or schools of astrology originating in or associated with a

Thông tin xuất bản: Germany : Angewandte Chemie (International ed. in English) , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 689770

Mitophagy, particularly in the context of drugs that disrupt mitochondrial membrane potential (MMP), represents a critical focus in pharmacology. However, the discovery and evaluation of MMP-disrupting drugs are often hampered using commercially available marker molecules that target similar or identical zones. These markers can significantly interfere with, obscure, or amplify the functional effects of MMP-targeting drugs, frequently leading to clinical failures. In response to this challenge, we propose a "one-two punch" drug design strategy that integrates both target-zone drug functionality and non-target zone biological reporting within a single small-molecule drug. We have developed a novel proof-of-concept mitophagy self-check drug (MitoSC) that exhibits dual-color and dual-localization properties. The functional component of this system is a variable MitoSC that disrupts mitochondrial membrane potential (MMP) homeostasis, thereby inducing mitophagy. Upon activation, this component transforms into a blue-fluorescent monomer (MitoSC-fun) specifically within the mitochondrial target zone. Concurrently, the biological reporting component is represented by a red-fluorescent monomer (MitoSC-rep) that localizes to lysosomes, the non-target zone. As mitophagy progresses, the fluorescent signals from MitoSC-rep (lysosomes) and MitoSC-fun (mitochondria) converge, enabling real-time monitoring of the mitophagic process. This strategy combines potent drug functionality with robust biological reporting, thereby minimizing interference and eliminating the complexities associated with external detection. Our findings underscore the potential of a single-molecule drug to exert target-zone specific actions while simultaneously providing non-target zone self-checking, offering a new perspective for drug design.
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