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. Over the last 20 years, antisense oligonucleotides (ASO) have emerged as a therapeutic platform for the treatment of genetic diseases but come with many challenges, including poor body-wide distribution, invasive delivery, and high dose side effects.
In this literature review, learn about the transcriptomic effects of combinatorial spinal muscular atrophy (SMA) therapies that used Dharmacon™ antisense oligonucleotides (ASO) and splice-modulating small molecules. Learn why the therapeutic approach of choice is targeting SMN2 and how combination treatment creates synergistic effects on SMN production while retaining minimal off-target effects.
Trust Revvity Dharmacon oligo synthesis experts for your custom ASO needs.
For research use only. Not for use in diagnostic procedures.