Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-nucleosidic Trivalent N-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo

We recently demonstrated that siRNAs conjugated to triantennary N‐acetylgalactosamine (GalNAc) induce robust RNAi‐mediated gene silencing in the liver, owing to uptake mediated by the asialoglycoprotein receptor (ASGPR). Novel monovalent GalNAc units, based on a non‐nucleosidic linker, were develope...

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Veröffentlicht in:	Chembiochem : a European journal of chemical biology 2015-04, Vol.16 (6), p.903-908
Hauptverfasser:	Rajeev, Kallanthottathil G., Nair, Jayaprakash K., Jayaraman, Muthusamy, Charisse, Klaus, Taneja, Nate, O'Shea, Jonathan, Willoughby, Jennifer L. S., Yucius, Kristina, Nguyen, Tuyen, Shulga-Morskaya, Svetlana, Milstein, Stuart, Liebow, Abigail, Querbes, William, Borodovsky, Anna, Fitzgerald, Kevin, Maier, Martin A., Manoharan, Muthiah
Format:	Artikel
Sprache:	eng
Schlagworte:	Acetylgalactosamine - chemistry Animals asialoglycoprotein receptor (ASGPR) Drug Carriers - chemistry Gene Silencing Hepatocytes - metabolism Mice N-acetylgalactosamine (GalNAc) oligonucleotide conjugates Prealbumin - deficiency Prealbumin - genetics RNA, Small Interfering - chemistry RNA, Small Interfering - genetics RNAi siRNA
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