发明名称 IN VIVO POLYNUCLEOTIDE DELIVERY CONJUGATES HAVING ENZYME SENSITIVE LINKAGES
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.
申请公布号 KR20130136494(A) 申请公布日期 2013.12.12
申请号 KR20137017195 申请日期 2011.12.28
申请人 ARROWHEAD RESEARCH CORPORATION 发明人 ROZEMA DAVID B.;LEWIS DAVID L.;WAKEFIELD DARREN H.;KITAS ERIC A.;HADWIGER PHILIPP;WOLFF JON A.;ROEHL INGO;MOHR PETER;HOFFMANN TORSTEN;JAHN HOFMANN KERSTIN;MUELLER HANS MARTIN;OTT GUENTHER;BLOKHIN ANDREI V.;CARLSON JEFFREY C.;BENSON JONATHAN D.
分类号 C07C233/05;A61K48/00;C07C69/96;C08G63/91 主分类号 C07C233/05
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