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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2014
Materias:
26
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4117763/
https://www.ncbi.nlm.nih.gov/pubmed/24992960
http://dx.doi.org/10.1093/nar/gku531

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