BENSON JONATHAN D,CARLSON JEFFREY C,BLOKHIN ANDREI V,OTT GUENTHER,MUELLER HANS MARTIN,JAHN-HOFMANN KERSTIN,ROEHL INGO,KITAS ERIC A,HADWIGER PHILIPP,LEWIS DAVID L,WOLFF JON A,WAKEFIELD DARREN H,ROZEMA,ROZEMA DAVID B,MOHR PETER,HOFFMANN TORSTEN
申请号:
ZA201302896
公开号:
ZA201302896B
申请日:
2013.04.22
申请国别(地区):
ZA
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 poiyamines 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 covalentiy linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.