What is an ASO?

An antisense oligonucleotide is a single stranded DNA oligo, complementary to an mRNA target. When added to the cell, the ASO hybridizes to the target mRNA resulting in a short double-stranded section.

ASOs can be used to block the production of proteins and are currently being studied in the treatment of several disease types. As of 2020, more than 50 antisense oligonucleotides were in clinical trials.