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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2017
Materias:
Chemical Biology and Nucleic Acid Chemistry
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5389643/
https://www.ncbi.nlm.nih.gov/pubmed/28158620
http://dx.doi.org/10.1093/nar/gkx060

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5389643/
https://www.ncbi.nlm.nih.gov/pubmed/28158620
http://dx.doi.org/10.1093/nar/gkx060

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