Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice

Triantennary N-acetyl galactosamine (GalNAc, GN3), a high-affinity ligand for the hepatocyte-specific asialoglycoprotein receptor (ASGPR), enhances the potency of second-generation gapmer antisense oligonucleotides (ASOs) 6–10-fold in mouse liver. When combined with next-generation ASO designs compr...

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Main Authors: Prakash, Thazha P., Graham, Mark J., Yu, Jinghua, Carty, Rick, Low, Audrey, Chappell, Alfred, Schmidt, Karsten, Zhao, Chenguang, Aghajan, Mariam, Murray, Heather F., Riney, Stan, Booten, Sheri L., Murray, Susan F., Gaus, Hans, Crosby, Jeff, Lima, Walt F., Guo, Shuling, Monia, Brett P., Swayze, Eric E., Seth, Punit P.
Format: Artigo
Jezik:Inglês
Izdano: Oxford University Press 2014
Teme:
26
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC4117763/
https://ncbi.nlm.nih.gov/pubmed/24992960
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gku531
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