Elucidation of the Biotransformation Pathways of a Galnac(3)-conjugated Antisense Oligonucleotide in Rats and Monkeys

Triantennary N-acetyl galactosamine (GalNAc(3)) is a high-affinity ligand for hepatocyte-specific asialoglycoprotein receptors. Conjugation with GalNAc(3) via a trishexylamino (THA)-C6 cluster significantly enhances antisense oligonucleotide (ASO) potency. Herein, the biotransformation, disposition,...

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Detalhes bibliográficos
Publicado no:Mol Ther Nucleic Acids
Main Authors: Shemesh, Colby S, Yu, Rosie Z, Gaus, Hans J, Greenlee, Sarah, Post, Noah, Schmidt, Karsten, Migawa, Michael T, Seth, Punit P, Zanardi, Thomas A, Prakash, Thazha P, Swayze, Eric E, Henry, Scott P, Wang, Yanfeng
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5014515/
https://ncbi.nlm.nih.gov/pubmed/27164023
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2016.31
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