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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...
Autores principales: | 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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7038945/ https://www.ncbi.nlm.nih.gov/pubmed/31980820 http://dx.doi.org/10.1093/nar/gkaa031 |
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