Mesyl phosphoramidate oligonucleotides as potential splice-switching agents: impact of backbone structure on activity and intracellular localization

A series of 2′-deoxy and novel 2′-O-methyl and 2′-O-(2-methoxyethyl) (2′-MOE) oligonucleotides with internucleotide methanesulfonyl (mesyl, μ) or 1-butanesulfonyl (busyl, β) phosphoramidate groups has been synthesized for evaluation as potential splice-switching oligonucleotides. Evaluation of their...

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מידע ביבליוגרפי
Main Authors: Hammond, SM, Sergeeva, OV, Melnikov, PA, Goli, L, Stoodley, J, Zatsepin, TS, Stetsenko, DA, Wood, MJA
פורמט: Journal article
שפה:English
יצא לאור: Mary Ann Liebert 2021