St. Jude Children’s Research Hospital shared on LinkedIn:
“See how St. Jude researchers use antisense oligonucleotides (ASOs) to address rare inherited genetic diseases.
Rare inherited diseases often expose one of the central challenges in precision medicine: a single gene may be linked to one condition, but many different mutations in that gene can produce the same disease.
That complexity can make clinical investigation more difficult and limit available treatment strategies.
Antisense oligonucleotides, or ASOs, offer a promising way to address that problem. By targeting RNA, ASOs can be designed to change how genetic information is processed before a harmful protein is made.
This creates a more adaptable framework for addressing disease-causing variants.
The broader significance is in what can be carried forward from one ASO to the next.
Design principles established for one mutation may help guide development for other variants in the same gene, helping move personalized therapies for rare inherited diseases closer to routine clinical reality.
As of this year, only 14 ASOs have been approved by the Food and Drug Administration for the treatment of a variety of diseases.
St. Jude is leading the design and use of ASOs to address rare inherited pediatric diseases where conventional approaches have fallen short.”
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