ANDREI V. BLOKHIN,DARREN H. WAKEFIELD,DAVID B. ROZEMA,DAVID L. LEWIS,ERIC A. KITAS,GUENTHER OTT,HANS MARTIN MUELLER,INGO ROEHL,JEFFREY C. CARLSON,JON A. WOLFF,JONATHAN D. BENSON,KERSTIN JAHN-HOFMANN,P
申请号：
BR112013014115
公开号：
BR112013014115A2
申请日:
2011.12.28
申请国别(地区):
BR
年份:
2019
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.
