J Clin Invest. 2023 Jun 1;133(11):e168554. doi: 10.1172/JCI168554.
Neurons are markedly compartmentalized, which makes them reliant on axonal transport to maintain their health. Axonal transport is important for anterograde delivery of newly synthesized macromolecules and organelles from the cell body to the synapse and for the retrograde delivery of signaling endosomes and autophagosomes for degradation. Dysregulation of axonal transport occurs early in neurodegenerative diseases and plays a key role in axonal degeneration. Here, we provide an overview of mechanisms for regulation of axonal transport; discuss how these mechanisms are disrupted in neurodegenerative diseases including Alzheimer's disease, Parkinson's disease, Huntington's disease, hereditary spastic paraplegia, amyotrophic lateral sclerosis, and Charcot-Marie-Tooth disease; and discuss therapeutic approaches targeting axonal transport.
神经元具有明显的区室化特征,这使得它们依赖于轴突运输来维持其健康。轴突运输对于从细胞体向突触正向输送新合成的大分子和细胞器,以及对于逆向输送信号转导内体和自噬体进行降解都很重要。轴突运输的失调发生在神经退行性疾病的早期,并且在轴突变性中起关键作用。在这里,我们提供了对轴突运输调节机制的概述;讨论了这些机制在包括阿尔茨海默病、帕金森病、亨廷顿病、遗传性痉挛性截瘫、肌萎缩侧索硬化症和 Charcot-Marie-Tooth 病在内的神经退行性疾病中是如何被破坏的;并讨论了针对轴突运输的治疗方法。