Rac1 inhibition prevents axonal cytoskeleton dysfunction in Transthyretin Amyloid Polyneuropathy
Joana Magalhães, Vítor Pacheco Dias, Jessica Eira, Marina I. Oliveira da Silva, Guilherme Nóvoa, Margarida Martins, Hung‐Wei Kan, Andreia Dias, Estefânia Carvalho, Mariana Santos, Ana Isabel Seixas, Teresa Coelho, Ricardo Jorge Ferreira Taipa, Joana Paes de Faria, Carolina Lemos, , Márcia A. Liz
This study shows that Rac1 inhibition prevents cytoskeletal dysfunction in ATTRv-PN by rescuing axonal defects in mice and supporting its therapeutic potential in patients.
Cell Reports · Oct 1, 2025Peer-reviewedNeuroscience