An LNA-amide modification that enhances the cell uptake and activity of phosphorothioate exon-skipping oligonucleotides

An LNA-amide modification that enhances the cell uptake and activity of phosphorothioate exon-skipping oligonucleotides

Oligonucleotides targeting mRNA are promising therapeutic agents but suffer from poor bioavailability. Here, the authors develop reduced-charge oligonucleotides with artificial LNA-amide linkages with improved cell uptake and minimal structural deviation to the DNA:RNA duplex.

Bibliographic Details
Main Authors: Ysobel R. Baker, Cameron Thorpe, Jinfeng Chen, Laura M. Poller, Lina Cox, Pawan Kumar, Wooi F. Lim, Lillian Lie, Graham McClorey, Sven Epple, Daniel Singleton, Michael A. McDonough, Jack S. Hardwick, Kirsten E. Christensen, Matthew J. A. Wood, James P. Hall, Afaf H. El-Sagheer, Tom Brown
Format: Article
Language:English
Published: Nature Portfolio 2022-07-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-022-31636-2

Internet

https://doi.org/10.1038/s41467-022-31636-2

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