Motor neurons have an extreme polarized morphology and heavily rely on efficient cargo transport along axons to maintain their neuronal connections and connections with muscles. Axonal transport deficits have been observed in almost all model systems of ALS. More and more studies have confirmed the close genetic and mechanistic linkage between axonal transport deficits with ALS pathogenesis. Moreover, several therapeutic approaches have been developed to target axonal transport deficits in ALS and showed promising effects in disease models. In this concise chapter, we summarize some major discoveries of axonal transport deficits in ALS pathogenesis and some related therapeutic strategies. We propose that targeting axonal transport may provide a potential therapeutic avenue for ALS.
Part of the book: Amyotrophic Lateral Sclerosis