Making Sense of Antisense Oligonucleotides

How does a potential drug discovered in the lab ultimately end up in people? We tackle this question in the context of exciting gene-modifying therapies called antisense oligonucleotides (ASOs). In this episode, we speak with Dr. Tim Miller to break down the science behind ASOs and learn more about his work in finding a cure for a genetic form of amyotrophic lateral sclerosis (ALS).

This episode was written and produced by Nancy Cai, Devika Nair, and Arja Ray.

Music used in this episode: “Thannoid”, “Bundt”, “Lupi”, “Partly Sage”, “Beignet”, “Trailrunner”, “Game Hens”, “Lord Weasel”, “The Zeppelin”, “Dorica”, “Our Fingers Cold”, “Gaena” by Blue Dot Sessions.

Cover image by Qualit Design.

For more information about spinal muscular atrophy (SMA), check out the spinal muscular dystrophy association website: www.mda.org/disease/spinal-muscular-atrophy.

There is also this great animation that shows how the Spinraza (nusinersen) ASO works in the body: www.spinraza.com/en_us/home/takin…nraza-works.html.

To read the results from the first ASO for Huntington’s disease, check out the New England Journal of Medicine article here: www.nejm.org/doi/full/10.1056/N…at=cr_pub%3Dpubmed.


Osama Ahmed