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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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Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
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