Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes

Antisense oligonucleotide (ASO) therapeutics show tremendous promise for the treatment of previously intractable human diseases but to exert their effects on cellular RNA processing they must first cross the plasma membrane by endocytosis. The conjugation of ASOs to a receptor ligand can dramaticall...

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Main Authors: Michael Tanowitz, Lisa Hettrick, Alexey S. Revenko, Garth A. Kinberger, Thazha P. Prakash, Punit P. Seth
Formato: Artigo
Idioma:inglés
Publicado: 2017
Acceso en liña:https://doi.org/10.1093/nar/gkx960
https://academic.oup.com/nar/article-pdf/45/21/12388/22146518/gkx960.pdf
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