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InFUSing Antisense Oligonucleotides for Treating ALS

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Journal Trends Mol Med
Date 2022 Mar 5
PMID 35246398
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Abstract

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.

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