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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2022
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Materias: | |
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 |
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