Site-specific replacement of phosphorothioate with alkyl phosphonate linkages enhances the therapeutic profile of gapmer ASOs by modulating interactions with cellular proteins

Phosphorothioate-modified antisense oligonucleotides (PS-ASOs) interact with a host of plasma, cell-surface and intracellular proteins which govern their therapeutic properties. Given the importance of PS backbone for interaction with proteins, we systematically replaced anionic PS-linkages in toxic...

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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: Migawa, Michael T, Shen, Wen, Wan, W Brad, Vasquez, Guillermo, Oestergaard, Michael E, Low, Audrey, De Hoyos, Cheryl L, Gupta, Ruchi, Murray, Susan, Tanowitz, Michael, Bell, Melanie, Nichols, Joshua G, Gaus, Hans, Liang, Xue-hai, Swayze, Eric E, Crooke, Stanley T, Seth, Punit P
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2019
Assuntos:
NAR Breakthrough Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6582325/
https://ncbi.nlm.nih.gov/pubmed/31034558
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkz247
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