ALS/FTD-Linked Mutation in FUS Suppresses Intra-axonal Protein Synthesis and Drives Disease Without Nuclear Loss-of-Function of FUS

Mutations in FUS are causative of ALS and frontotemporal dementia (FTD). López-Erauskin et al. show that disease-causing mutant FUS inhibits intra-axonal protein synthesis and provokes hippocampal synaptic loss and dysfunction without loss of nuclear FUS function or FUS aggregation.