申请人:Protiva Biotherapeutics, Inc.
公开号:US20140134260A1
公开(公告)日:2014-05-15
The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid in vivo. The compositions of the present invention are highly potent, thereby allowing effective know-down of a specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.
本发明提供了一种用于向细胞传递治疗剂的组合物和方法。特别地,这些包括新颖的阳离子脂质和核酸-脂质颗粒,它们提供了核酸的高效封装和体内高效传递。本发明的组合物非常有效,因此可以在相对较低的剂量下有效地知道一个特定的靶蛋白。此外,本发明的组合物和方法比以前在艺术上已知的组合物和方法毒性更小,提供了更大的治疗指数。