Antisense Oligonucleotides for the Study and Treatment of ALS

Benjamin D. Boros, Kathleen M. Schoch, Collin J. Kreple, Timothy Miller

Research output: Contribution to journalReview articlepeer-review

3 Scopus citations

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by motor neuron loss. ALS is now associated with mutations in numerous genes, many of which cause disease in part through toxic gain-of-function mechanisms. Antisense oligonucleotides (ASOs) are small sequences of DNA that can reduce expression of a target gene at the post-transcriptional level, making them attractive for neutralizing mutant or toxic gene products. Advancements in the medicinal chemistries of ASOs have improved their pharmacodynamic profile to allow safe and effective delivery to the central nervous system. ASO therapies for ALS have rapidly developed over the last two decades, and ASOs that target SOD1, C9orf72, FUS, and ATXN2 are now in clinical trials for familial or sporadic forms of ALS. This review discusses the current state of ASO therapies for ALS, outlining their successes from preclinical development to early clinical trials.

Original languageEnglish
Pages (from-to)1145-1158
Number of pages14
JournalNeurotherapeutics
Volume19
Issue number4
DOIs
StatePublished - Jul 2022

Keywords

  • Amyotrophic lateral sclerosis
  • Antisense oligonucleotide
  • Clinical trials
  • Motor neuron disease
  • Therapy

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