Failure of C9orf72 sense repeat-targeting antisense oligonucleotides: lessons learned and the path forward

Brain. 2024 Aug 1;147(8):2607-2609. doi: 10.1093/brain/awae168.
No abstract available

Publication types

  • Comment

MeSH terms

  • Amyotrophic Lateral Sclerosis / drug therapy
  • Amyotrophic Lateral Sclerosis / genetics
  • C9orf72 Protein* / genetics
  • DNA Repeat Expansion
  • Humans
  • Oligonucleotides, Antisense* / therapeutic use
  • Proteins* / genetics
  • Treatment Failure

Substances

  • C9orf72 Protein
  • Oligonucleotides, Antisense
  • C9orf72 protein, human
  • Proteins