Rac1 inhibition prevents axonal cytoskeleton dysfunction in transthyretin amyloid polyneuropathy.

Magalhães et al. reveal that Rac1 hyperactivation disrupts axonal cytoskeleton and transport in a mouse model of transthyretin amyloid polyneuropathy. They show that Rac1 inhibition prevents axonal degeneration and identify a patient-associated RACGAP1 variant suggesting a neuroprotective role for Rac1 inactivation.