St. Jude Family of Websites
Explore our cutting edge research, world-class patient care, career opportunities and more.
St. Jude Children's Research Hospital Home
- Fundraising
St. Jude Family of Websites
Explore our cutting edge research, world-class patient care, career opportunities and more.
St. Jude Children's Research Hospital Home
- Fundraising
Antisense<br>oligonucleotides illustrated
As our understanding of the origins of genetic diseases expands, so too does the ability to treat them with antisense oligonucleotides.
A paradigm shift is underway in how genetic diseases are treated, with individualized therapies that target the root cause of disease becoming an increasingly important part of the treatment landscape. While gene editing offers the ability to repair mutated genes at the source, another class of personalized therapy called antisense oligonucleotides (ASOs) offers a more flexible approach with equally impactful results. As of 2026, 14 ASO drugs have been approved by the Food and Drug Administration (FDA) for the treatment of a variety of neurologic and other diseases.
St. Jude is leading the design and use of ASOs to address rare inherited pediatric diseases where conventional approaches have fallen short. This effort will ensure children affected by these diseases have access to personalized therapies that can stop, or potentially reverse, the trajectory of their conditions, offering hope where few options previously existed.
Rare genetic diseases arise from inherited or new genetic changes.
The likelihood of this occurring is low across a population, but it is isolating and devastating for families when it does happen.
While individually rare, collectively thousands of rare genetic diseases affect millions of individuals.
While many rare inherited diseases are associated with a single gene, a number of mutations within this gene can lead to the same disease, often with a wide range of severities and outlooks.
This means each instance may result in a similar pathogenic impact but is different enough to require an individualized approach.
To address this, researchers are exploring ways to target messenger RNA (mRNA), the intermediate molecule transcribed from DNA, which is used as a blueprint to produce a protein.
mRNA carries the same sequence as the coding, or “sense” strand of the gene, including the disease-causing error.
The mRNA is consequently translated into a disease-causing, mutated protein or can result in a deficiency of a necessary protein.
Scientists have identified ways to alter mRNA using a short strand of nucleotides called an oligonucleotide.
These oligonucleotides are designed to be complementary to the mRNA, hence, are called “antisense” oligonucleotides.
ASOs alter how the mRNA is processed to either remove the mRNA completely, preventing the mutated protein from being produced, or compensate for the mutation, resulting in a partially or fully corrected protein.
While ASOs are designed to correct for a specific mutation, the knowledge gained from the design of one ASO can also help guide similar approaches for patients with a different mutation within the same gene.
This can reduce the amount of time needed to clear regulatory hurdles and make the road to personalized care for rare inherited diseases fully realizable.
As our understanding of the origins of genetic diseases expands, so too does the ability to treat them with ASOs. These therapies hold the promise to treat not just rare inherited diseases, but many diseases rooted in genetics.
What was once a life-altering fate written in genetic code can now be rewritten, restoring hope once thought out of reach for thousands of children with catastrophic genetic diseases.
About the authors
