Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides

Therapeutic oligonucleotides are often modified using the phosphorothioate (PS) backbone modification which enhances stability from nuclease mediated degradation. However, substituting oxygen in the phosphodiester backbone with sulfur introduce chirality into the backbone such that a full PS 16-mer...

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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: Østergaard, Michael E, De Hoyos, Cheryl L, Wan, W Brad, Shen, Wen, Low, Audrey, Berdeja, Andres, Vasquez, Guillermo, Murray, Susan, Migawa, Michael T, Liang, Xue-hai, Swayze, Eric E, Crooke, Stanley T, Seth, Punit P
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2020
Assuntos:
Chemical Biology and Nucleic Acid Chemistry
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7038945/
https://ncbi.nlm.nih.gov/pubmed/31980820
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkaa031
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