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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Detaylı Bibliyografya
Yayımlandı:Nucleic Acids Res
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Oxford University Press 2017
Konular:
Chemical Biology and Nucleic Acid Chemistry
Online Erişim: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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