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Comment
. 2022 Apr;28(4):253-254.
doi: 10.1016/j.molmed.2022.02.006. Epub 2022 Mar 1.

InFUSing antisense oligonucleotides for treating ALS

Philippe Codron  1 , Julien Cassereau  2 , Patrick Vourc'h  3
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InFUSing antisense oligonucleotides for treating ALS

Philippe Codron et al. Trends Mol Med. 2022 Apr.

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.

Trial registration: ClinicalTrials.gov NCT04768972.

Keywords: FUS; TDP-43; amyotrophic lateral sclerosis; antisense oligonucleotide.

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