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...

Descrizione completa

Salvato in:
Dettagli Bibliografici
Pubblicato in:Nucleic Acids Res
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Oxford University Press 2017
Soggetti:
Chemical Biology and Nucleic Acid Chemistry
Accesso online: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
Tags: Aggiungi Tag
Nessun Tag, puoi essere il primo ad aggiungerne! !

Documenti analoghi