Overriding FUS autoregulation in mice triggers gain-of-toxic dysfunctions in RNA metabolism and autophagy-lysosome axis
Mutations in coding and non-coding regions of FUS cause amyotrophic lateral sclerosis (ALS).The latter mutations may exert toxicity by increasing FUS accumulation.We show here that broad expression within the nervous system of wild-type or either of two ALS-linked mutants of human FUS in mice produces progressive motor phenotypes accompanied by cha