ASO67 is made up of 20 nucleotides that follow the Watson-Crick base pairing, binding to the targeted region. It’s composed of a DNA centre and RNA flanking regions, allowing it to effectively bind to the SNP region and be recognised by RNase H. We chemically modified ASO67 on its RNA backbone, nucleobase, and ribose sugar to make it more “drug-like” for optimised drug delivery to the central nervous system and less susceptible to nuclease degradation. Scroll to see more details about the chemical modification.