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Expanding the toolbox: Emerging antisense oligonucleotide mechanisms for modulating gene expression
Isabella Trew1,2, Steve D Wilton1,2, Jessica M Cale1,2
1Personalised Medicine Centre, Health Futures Institute, Murdoch University, Perth, WA 6150, Australia.
Molecular Therapy. Nucleic Acids
|June 4, 2026
Summary
Antisense oligonucleotides (ASOs) are versatile therapeutics targeting genetic sequences. Advances enable ASOs to modulate diverse molecular processes, expanding their potential for treating various conditions.
Area of Science:
- Molecular Medicine
- RNA Biology
- Genomics
Background:
- Antisense oligonucleotides (ASOs) are established in molecular medicine.
- Advances in synthesis, chemical modification, and genomic understanding have improved ASO therapeutics.
- ASOs offer high specificity and tailorability for various conditions.
Purpose of the Study:
- To highlight the evolution and expanding mechanisms of ASOs in therapeutics.
- To emphasize the need for a holistic design approach considering molecular mechanisms.
- To showcase the potential of ASOs in modulating diverse RNA processing events.
Main Methods:
- Review of decades of research on ASO synthesis and chemical modifications.
- Analysis of advancements in understanding the human genome and transcriptome.
- Exploration of emerging research on ASO mechanisms of action.
Main Results:
- ASOs have evolved from simple gene expression inhibitors to versatile modulators.
- Effective ASO design requires a holistic approach considering molecular mechanisms.
- ASOs can modulate numerous pre-mRNA processing events, including splicing, polyadenylation, microRNA activity, and translation.
Conclusions:
- The therapeutic potential of ASOs is significantly broader than initially recognized.
- A deeper understanding of RNA biology continues to expand ASO therapeutic applications.
- ASO-based therapeutics can benefit a wider range of conditions and patients by targeting diverse molecular processes.
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