肌萎缩侧索硬化症(amyotrophic lateral sclerosis,ALS)是目前无有效治疗的一种神经退行性疾病，其发病机制不清。近期研究发现反式激活应答 DNA 结合蛋白 43(transactive response element DNA binding protein-43,TDP-43)在包括 ALS、额颞叶痴呆等神经退行性疾病中起重要作用。本文总结了近 10 年来对 TDP-43 结构的最新认识及其在神经退行性疾病发病机制的作用与潜在的治疗靶点，以期为中西医结合诊治 ALS 提供一定的基础研究依据。

Amyotrophic lateral sclerosis (ALS)is a neurodegenerative disease that currently has no effective treatment, and its pathogenesis is unclear. Recent studies have found that transactive response element DNA binding protein-43 (TDP-43) plays an important role in neurodegenerative diseases including ALS and frontotemporal dementia. This article summarizes the latest understanding of the structure of TDP-43 in the past decade, its role in the pathogenesis of neurodegenerative diseases, and potential therapeutic targets, in order to provide a basic research basis for the integrated diagnosis and treatment of ALS with Chinese and Western medicine.