An LNA-amide modification that enhances the cell uptake and activity of phosphorothioate exon-skipping oligonucleotides.
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.
Baker YR, et al. Among authors: singleton d.
Nat Commun. 2022 Jul 12;13(1):4036. doi: 10.1038/s41467-022-31636-2.
Nat Commun. 2022.
PMID: 35821218
Free PMC article.