Antisense oligonucleotide therapies for Amyotrophic Lateral Sclerosis: Existing and emerging targets

Joseph R. Klim, Caroline Vance, Emma L. Scotter*

*Corresponding author for this work

Research output: Contribution to journalShort surveypeer-review

21 Citations (Scopus)
688 Downloads (Pure)


Amyotrophic lateral sclerosis (ALS) is a disease with highly heterogenous causes, most of which remain unknown, a multitude of possible disease mechanisms, and no therapy currently available that can halt disease progression. However, recent advances in antisense oligonucleotides have made them a viable option for targeted therapeutics for patients. These molecules offer a method of targeting RNA that is highly specific, adaptable, and does not require viral delivery. Antisense oligonucleotides are therefore being developed for several genetic causes of ALS. Furthermore, biological pathways involved in the pathogenesis of disease also offer tantalizing targets for intervention using antisense oligonucleotides. Here we detail existing and potential targets for antisense oligonucleotides in ALS and briefly examine the requirements for these drugs to reach and be effective in clinic.

Original languageEnglish
Pages (from-to)149-153
Number of pages5
JournalInternational Journal of Biochemistry and Cell Biology
Early online date20 Mar 2019
Publication statusPublished - May 2019


  • Amyotrophic lateral sclerosis
  • Antisense oligonucleotides
  • Molecular medicine
  • Neurodegenerative diseases

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