ROEHL INGO,ROZEMA DAVID B.,LEWIS DAVID L.,KITAS ERIC A.,HADWIGER PHILIPP,WOLFF JON A.,HOFFMANN TORSTEN,JAHN-HOFMANN KERSTIN,MUELLER HANS MARTIN,OTT GUENTHER,BLOKHIN ANDREI V.,BENSON JONATHAN D.,MOHR P
申请号：
NZ61165611
公开号：
NZ611656A
申请日:
2011.12.28
申请国别(地区):
NZ
年份:
2014
代理人:
摘要：
The disclosure relates to compounds for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compounds comprise amphipathic membrane active polyamines comprising a steric stabiliser or targeting ligand covalently linked to a dipeptide amidobenzyl-carbonate. The compound may take the form of the structure in the abstract figure where the groups are as defined in the specification. The steric stabiliser may be polyethylene glycol and the targeting ligand may be an asialoglycoprotein receptor (ASGPr) ligand. The disclosure also relates to a delivery polymer and methods for delivering the RNAi polynucleotide to a cell using the compounds.
