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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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5014515/
https://www.ncbi.nlm.nih.gov/pubmed/27164023
http://dx.doi.org/10.1038/mtna.2016.31