ALS-related FUS mutations alter axon growth in motoneurons and affect HuD/ELAVL4 and FMRP activity

Abstract

Maria Giovanna Garone et al. use iPSC and mouse models to evaluate a mechanistic link between aberrant axonal phenotypes in ALS and the alteration of a cross-regulatory circuitry involving three RNA binding proteins: FUS, HuD and FMRP. Their results suggest NRN1 as a potential therapeutic target for ALS and provide further insight into the pathogenesis of this critical disorder.