Elucidation of the Biotransformation Pathways of a Galnac3-conjugated Antisense Oligonucleotide in Rats and Monkeys

Elucidation of the Biotransformation Pathways of a Galnac3-conjugated Antisense Oligonucleotide in Rats and Monkeys

Triantennary N-acetyl galactosamine (GalNAc3) is a high-affinity ligand for hepatocyte-specific asialoglycoprotein receptors. Conjugation with GalNAc3 via a trishexylamino (THA)-C6 cluster significantly enhances antisense oligonucleotide (ASO) potency. Herein, the biotransformation, disposition, and...

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Bibliographic Details
Main Authors: Colby S Shemesh, Rosie Z Yu, Hans J Gaus, Sarah Greenlee, Noah Post, Karsten Schmidt, Michael T Migawa, Punit P Seth, Thomas A Zanardi, Thazha P Prakash, Eric E Swayze, Scott P Henry, Yanfeng Wang
Format: Article
Language:English
Published: Elsevier 2016-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense oligonucleotide
biotransformation
metabolism
N-acetyl galactosamine
targeted drug delivery
Online Access:http://www.sciencedirect.com/science/article/pii/S216225311730046X

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http://www.sciencedirect.com/science/article/pii/S216225311730046X

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