申请人:Heyes James
公开号:US20120172411A1
公开(公告)日:2012-07-05
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.
本发明提供了用于将治疗剂传递至细胞的组合物和方法。具体包括新型阳离子脂质和核酸-脂质颗粒,可有效地封装核酸并有效地将封装的核酸传递至体内的细胞。本发明的组合物非常强效,因此可以在相对较低的剂量下有效地降低特定靶蛋白的表达。此外,本发明的组合物和方法与先前已知的组合物和方法相比,毒性更低,并提供更大的治疗指数。