申请人:Martin Alan
公开号:US20130064894A1
公开(公告)日:2013-03-14
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 to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-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.
本发明提供了递送治疗剂到细胞的组合物和方法。特别是,这些包括新型阳离子脂质和核酸-脂质颗粒,它们提供了核酸的高效封装和封装的核酸在体内的高效递送到细胞。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抑制特定目标蛋白。此外,与先前已知的组合物和方法相比,本发明的组合物和方法毒性更小,提供更大的治疗指数。