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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: 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.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Oxford University Press 2014
Pynciau:
26
Mynediad Ar-lein: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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