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FUScinating insights into motor neuron degeneration
Authors:Dorothee Dormann
Affiliation:1. BioMedical Center (BMC), Ludwig‐Maximilians‐Universit?t München, Planegg‐Martinsried, Germany;2. Munich Cluster for Systems Neurology (SyNergy), Munich, Germany;3. Graduate School of Systemic Neurosciences (GSN), Planegg‐Martinsried, Germany
Abstract:Point mutations in FUS cause amyotrophic lateral sclerosis (ALS), a devastating neurodegenerative disease—but do they do that by a loss of the protein's normal function, or by endowing it with novel toxic functions, or both? In this issue of The EMBO Journal, Scekic‐Zahirovic et al ( 2016 ) report that mutant FUS, but not the complete loss of FUS, triggers motor neuron degeneration in mice, arguing for a toxic gain‐of‐function mechanism.
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