公开(公告)号：US20130121954A1

公开(公告)日：2013-05-16

申请号：US13592393

申请日：2012-08-23

申请人： Darren H. Wakefield ,  Nicholas A. Rossi ,  Dan Sheik

发明人： Darren H. Wakefield ,  Nicholas A. Rossi ,  Dan Sheik

IPC分类号： A61K47/48

CPC分类号： A61K47/48176 ,  A61K31/7105 ,  A61K47/58 ,  C08F218/04 ,  C08F218/10 ,  C08F2218/22 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/351 ,  C12N2320/30

摘要： The present invention is directed membrane active poly(vinyl ester) polymers and compositions for targeted delivery of RNA interference (RNAi) polynucleotides to cells in vivo. RNAi polynucleotides are conjugated to the poly(vinyl ester) polymers and the polymers are reversibly modified to enable in vivo targeted delivery. Membrane activity of the poly(vinyl ester) provides for movement of the RNAi polynucleotides from outside the cell to inside the cell. Reversible modification provides physiological responsiveness.

公开(公告)号：US08932572B2

公开(公告)日：2015-01-13

申请号：US13592393

申请日：2012-08-23

申请人： Darren H. Wakefield ,  Nicholas Rossi ,  Dan Sheik

发明人： Darren H. Wakefield ,  Nicholas Rossi ,  Dan Sheik

IPC分类号： A61K31/74 ,  A61K47/48 ,  C08F226/02 ,  C08F218/04

CPC分类号： A61K47/48176 ,  A61K31/7105 ,  A61K47/58 ,  C08F218/04 ,  C08F218/10 ,  C08F2218/22 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/351 ,  C12N2320/30

摘要： The present invention is directed membrane active poly(vinyl ester) polymers and compositions for targeted delivery of RNA interference (RNAi) polynucleotides to cells in vivo. RNAi polynucleotides are conjugated to the poly(vinyl ester) polymers and the polymers are reversibly modified to enable in vivo targeted delivery. Membrane activity of the poly(vinyl ester) provides for movement of the RNAi polynucleotides from outside the cell to inside the cell. Reversible modification provides physiological responsiveness.

摘要翻译： 本发明涉及膜活性聚（乙烯基酯）聚合物和用于将RNA干扰（RNAi）多核苷酸靶向递送至细胞体内的组合物。 RNAi多核苷酸与聚（乙烯基酯）聚合物缀合，并且可逆地修饰聚合物以实现体内靶向递送。 聚（乙烯基酯）的膜活性提供了RNAi多核苷酸从细胞外部到细胞内的运动。 可逆修饰提供生理反应。