Disruption of axonal transport would be a good target for his drug.
What is disruption of axonal transport?
- Axonal transport defects are among the early molecular events leading to neurodegeneration in mouse models of amyotrophic lateral sclerosis (ALS).
- Gene expression profiles indicate that dynactin-1 mRNA is downregulated in degenerating spinal motor neurons of autopsied patients with sporadic ALS.
- Disruption of axonal transport also underlies the pathogenesis of spinal muscular atrophy and hereditary spastic paraplegias.
- The neurotoxin β,β′-iminodipropionitrile (IDPN) selectively disrupts slow axonal transport without affecting fast anterograde or retrograde axonal transport.
- Impairment of slow axonal transport causes a proximal accumulation of neurofilaments.
- Because neurofilaments regulate axonal diameter, this accumulation leads to a marked swelling of the axon.
- Acrylamide causes decreased axonal transport, also causing proximal accumulations of neurofilaments and swelling.
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