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

Oligonucleotides that target mRNA have great promise as therapeutic agents for life-threatening conditions but suffer from poor bioavailability, hence high cost. As currently untreatable diseases come within the reach of oligonucleotide therapies, new analogues are urgently needed to address this. W...

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Detalles Bibliográficos
Autores principales: Baker, Ysobel R., Thorpe, Cameron, Chen, Jinfeng, Poller, Laura M., Cox, Lina, Kumar, Pawan, Lim, Wooi F., Lie, Lillian, McClorey, Graham, Epple, Sven, Singleton, Daniel, McDonough, Michael A., Hardwick, Jack S., Christensen, Kirsten E., Wood, Matthew J. A., Hall, James P., El-Sagheer, Afaf H., Brown, Tom
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9276774/
https://www.ncbi.nlm.nih.gov/pubmed/35821218
http://dx.doi.org/10.1038/s41467-022-31636-2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9276774/
https://www.ncbi.nlm.nih.gov/pubmed/35821218
http://dx.doi.org/10.1038/s41467-022-31636-2

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