Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor

Targeted delivery of antisense oligonucleotides (ASO) to hepatocytes via the asialoglycoprotein receptor (ASGR) has improved the potency of ASO drugs ∼30-fold in the clinic (1). In order to fully characterize the effect of GalNAc valency, oligonucleotide length, flexibility and chemical composition...

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Enregistré dans:
Détails bibliographiques
Publié dans:Nucleic Acids Res
Auteurs principaux: Schmidt, Karsten, Prakash, Thazha P., Donner, Aaron J., Kinberger, Garth A., Gaus, Hans J., Low, Audrey, Østergaard, Michael E., Bell, Melanie, Swayze, Eric E., Seth, Punit P.
Format: Artigo
Langue:Inglês
Publié: Oxford University Press 2017
Sujets:
Chemical Biology and Nucleic Acid Chemistry
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5389643/
https://ncbi.nlm.nih.gov/pubmed/28158620
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkx060
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