An LNA-amide modification that enhances the cell uptake and activity of phosphorothioate exon-skipping oligonucleotides
oleh: 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 |
|---|---|
| Diterbitkan: | Nature Portfolio 2022-07-01 |
Deskripsi
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.