The question of a loss or toxic gain of function in FUS-related amyotrophic lateral sclerosis is still debated. Recently, Korobeynikov et al. argued that FUS mutations lead to a gain of function and showed that lowering wild-type and mutant FUS levels could be a promising therapeutic strategy.
Trial registration: ClinicalTrials.gov NCT04768972.
Keywords: FUS; TDP-43; amyotrophic lateral sclerosis; antisense oligonucleotide.
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