RNase-H-mediated silencing in the CNS proves predictably nontrivial

Med. 2022 Nov 11;3(11):733-734. doi: 10.1016/j.medj.2022.10.004.

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease primarily affecting motor neurons. Recently,1 Miller et al. presented phase 3 data for the RNase-H-recruiting antisense oligonucleotide (ASO) targeting superoxide dismutase 1 (SOD1) in ALS, which represents a step forward in the deployment of ASO therapeutics against CNS targets.

Publication types

  • Comment

MeSH terms

  • Amyotrophic Lateral Sclerosis* / genetics
  • Humans
  • Neurodegenerative Diseases*
  • Oligonucleotides, Antisense / therapeutic use
  • Ribonuclease H
  • Superoxide Dismutase
  • Superoxide Dismutase-1 / genetics

Substances

  • Ribonuclease H
  • Superoxide Dismutase-1
  • Superoxide Dismutase
  • Oligonucleotides, Antisense