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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Main Authors: Baker, YR, Thorpe, C, Chen, J, Poller, LM, Cox, L, Kumar, P, Lim, WF, Lie, L, McClorey, G, Epple, S, Singleton, D, McDonough, MA, Hardwick, JS, Christensen, KE, Wood, MJA, Hall, JP, El-Sagheer, AH, Brown, T
格式: Journal article
语言:English
出版: Springer Nature 2022

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