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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Vydáno v:Nucleic Acids Res
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: Oxford University Press 2017
Témata:
Chemical Biology and Nucleic Acid Chemistry
On-line přístup: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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