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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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Bibliografische gegevens
Gepubliceerd in:Nucleic Acids Res
Hoofdauteurs: 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
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Oxford University Press 2020
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Online toegang: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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