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.
|Title of host publication||Translational Neuroscience|
|Subtitle of host publication||Fundamental Approaches for Neurological Disorders|
|Number of pages||11|
|State||Published - Jan 1 2016|