IN VIVO POLYNUCLEOTIDE DELIVERY CONJUGATES HAVING ENZYME SENSITIVE LINKAGES

Fecha de publicación: 24/10/2014
Fuente: WIPO "apis cerana"
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.