Antisense oligonucleotides (ASO) are short DNA-like chemicals that bind to RNA by Watson–Crick base pairing and modulate function of the RNA. These chemicals do not cross the blood brain barrier, but may be delivered directly to the cerebral spinal fl uid (CSF) to achieve widespread distribution throughout the brain and spinal cord. ASO have been used to target genes associated with familial amyotrophic lateral sclerosis (ALS), such as SOD1 and C9orf72 as well as miRNAs.A Phase I trial for SOD1-targeting ASO showed excellent safety and important pharmacodynamics. ASO are a promising therapeutic approach for ALS.

Original languageEnglish
Title of host publicationTranslational Neuroscience
Subtitle of host publicationFundamental Approaches for Neurological Disorders
PublisherSpringer US
Number of pages11
ISBN (Electronic)9781489976543
ISBN (Print)9781489976529
StatePublished - Jan 1 2016


  • ALS
  • C9orf72
  • SOD1
  • mRNA
  • miRNA


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