How Does It Work? | Antisense Oligonucleotide Therapy

In this week’s installment of our “How Does It Work?” video series, we’ll explore Antisense Oligonucleotide Therapy.

Errors in DNA, known as gene mutations, can originate harmful proteins that cause disease. However, we can target the mRNA involved in the formation of specific disease associated proteins by using drugs called antisense oligonucleotides.

They work by binding to specific sequences of nucleotides present within the mRNA structure. This can induce mechanisms that either decrease, restore, or modify protein expression. And since proteins are often linked to disease, there’s huge potential to treat a broad range of diseases with this technology.

This includes rare genetic disorders, cancers, and neurological and autoimmune disorders.