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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2019
Materias:
NAR Breakthrough Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6582325/
https://www.ncbi.nlm.nih.gov/pubmed/31034558
http://dx.doi.org/10.1093/nar/gkz247

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