InFUSing antisense oligonucleotides for treating ALS

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

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.

Publication types

  • Comment

MeSH terms

  • Amyotrophic Lateral Sclerosis* / genetics
  • Amyotrophic Lateral Sclerosis* / therapy
  • DNA-Binding Proteins / genetics
  • DNA-Binding Proteins / metabolism
  • Humans
  • Mutation
  • Oligonucleotides, Antisense / genetics
  • Oligonucleotides, Antisense / therapeutic use
  • RNA-Binding Protein FUS / genetics

Substances

  • DNA-Binding Proteins
  • Oligonucleotides, Antisense
  • RNA-Binding Protein FUS

Associated data

  • ClinicalTrials.gov/NCT04768972