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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Udgivet i:Nucleic Acids Res
Main Authors: 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
Sprog:Inglês
Udgivet: Oxford University Press 2017
Fag:
Chemical Biology and Nucleic Acid Chemistry
Online adgang: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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