Functional Mechanisms of Antisense Oligonucleotides (ASOs)

Description

The figure illustrates some of the functional mechanisms through which ASOs binding to their mRNA target ultimately leads to a reduction in the target protein. The panel on the left illustrates an ASO binding to an mRNA target, activating the RNase H degradation pathway, leading to digestion of the mRNA-ASO heteroduplex. The panel on the right shows an ASO binding to its target mRNA and inhibiting the initiation or the continuation of protein translation through steric hindrance.

Acknowledgements

References

Evers, M. M., Toonen, L. J., & van Roon-Mom, W. M. (2015). Antisense oligonucleotides in therapy for neurodegenerative disorders. Advanced drug delivery reviews, 87, 90-103. Benizri, S., Gissot, A., Martin, A., Vialet, B., Grinstaff, M. W., & Barthélémy, P. (2019). Bioconjugated oligonucleotides: recent developments and therapeutic applications. Bioconjugate chemistry, 30(2), 366-383. Collotta, D., Bertocchi, I., Chiapello, E., & Collino, M. (2023). Antisense oligonucleotides: a novel Frontier in pharmacological strategy. Frontiers in Pharmacology, 14, 1304342.

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