线粒体融合蛋白2:潜在的药物靶点  

Mitofusin 2:an emerging drug target

作　　者：帅育兵 王秋丹 何田玉 曹丽娟[1] SHUAI Yubing;WANG Qiudan;HE Tianyu;CAO Lijuan(State Key Laboratory of Natural Medicines,China Pharmaceutical University,Nanjing 211198,China)

机构地区：[1]中国药科大学多靶标天然药物全国重点实验室,南京211198

出　　处：《中国药科大学学报》2025年第1期1-9,共9页Journal of China Pharmaceutical University

基　　金：国家自然科学基金项目(No.82373886)。

摘　　要：线粒体融合蛋白2(mitofusin2,MFN2)定位于线粒体外膜,是参与线粒体融合以及维持线粒体形态的关键因子。由于MFN2在细胞内的功能多样性,其参与多种疾病进展,尤其在2型腓骨肌萎缩症中,以MFN2为靶点的药物研发正在成为热点。本文对MFN2的功能以及在多种疾病中的作用进行了回顾,并概述了针对MFN2的药物开发现现状,总结了目前进入临床前研究阶段的潜在药物分子,以期为MFN2作为靶点的药物研究以及疗法开发提供参考。Mitofusin 2(MFN2)residing on the outer mitochondrial membrane is a pivotal factor participating in mitochondrial fusion and maintaining mitochondrial morphology.Due to its multifaceted cellular functions,MFN2 is implicated in the pathogenesis of diverse maladies,notably type 2 Charcot-Marie-Tooth disease,which has catalyzed a surge in pharmaceutical endeavors directed towards MFN2.This article reviews the function of MFN2 and its role in a variety of diseases,outlines the current status of drug discovery against MFN2,and summarizes potential drug molecules currently in preclinical research,aiming to provide some reference for the research and development of drugs and therapies targeting MFN2.

关 键 词：线粒体融合蛋白2 功能 疾病机制 药物靶点 神经系统疾病 心血管疾病 

分 类 号：R915[医药卫生—微生物与生化药学]