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...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
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.
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2014
Assuntos:
26
Acesso em linha: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
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados