发明名称 Peptide-based in vivo siRNA delivery system
摘要 The present invention is directed compositions for targeted delivery of RNA interference (RNAi) polynucleotides to hepatocytes in vivo. Targeted RNAi polynucleotides are administered together with co-targeted melittin delivery peptides. Delivery peptides provide membrane penetration function for movement of the RNAi polynucleotides from outside the cell to inside the cell. Reversible modification provides physiological responsiveness to the delivery peptides.
申请公布号 US9107957(B2) 申请公布日期 2015.08.18
申请号 US201313926380 申请日期 2013.06.25
申请人 Arrowhead Madison Inc. 发明人 Rozema David B;Lewis David L;Wakefield Darren;Kitas Eric;Hadwiger Philipp;Wolff Jon;Röhl Ingo;Mohr Peter;Hoffmann Torsten;Jahn-Hofmann Kerstin;Mueller Hans Martin;Ott Günther;Blokhin Andrei V;Benson Jonathan D;Carlson Jeffrey C
分类号 C07H21/04;C07H21/02;C07K5/00;A61K47/48;C12N15/11 主分类号 C07H21/04
代理机构 代理人 Ekena Kirk
主权项 1. A reversibly masked melittin peptide having the structure represented by Melittin-(L-T)x wherein Melittin is a melittin peptide, L is a physiologically labile reversible linkage, T comprises a targeting ligand, and x is an integer greater than zero.
地址 Madison WI US