Site-specific incorporation of 5′-methyl DNA enhances the therapeutic profile of gapmer ASOs

We recently showed that site-specific incorporation of 2′-modifications or neutral linkages in the oligo-deoxynucleotide gap region of toxic phosphorothioate (PS) gapmer ASOs can enhance therapeutic index and safety. In this manuscript, we determined if introducing substitution at the 5′-position of...

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Bibliografiske detaljer
Udgivet i:Nucleic Acids Res
Main Authors: Vasquez, Guillermo, Freestone, Graeme C, Wan, W Brad, Low, Audrey, De Hoyos, Cheryl Li, Yu, Jinghua, Prakash, Thazha P, Ǿstergaard, Michael E, Liang, Xue-hai, Crooke, Stanley T, Swayze, Eric E, Migawa, Michael T, Seth, Punit P
Format: Artigo
Sprog:Inglês
Udgivet: Oxford University Press 2021
Fag:
Chemical Biology and Nucleic Acid Chemistry
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7913697/
https://ncbi.nlm.nih.gov/pubmed/33544849
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkab047
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