David B Rozema,David L Lewis,Darren H Wakefield,Eric A Kitas,Philipp Hadwiger,Jon A Wolff,Ingo Roehl,Peter Mohr,Torsten Hoffmann,Kerstin Jahn-Hofmann,Hans Martin Mueller,Guenther Ott,Andrei V Blokhin
申请号：
US13833814
公开号：
US08802773B2
申请日:
2013.03.15
申请国别(地区):
US
年份:
2014
代理人:
摘要：
The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active polyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalently linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.
