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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Publicat a:Mol Ther Nucleic Acids
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2016
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Original Article
Accés en línia: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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