申请人:TEKMIRA PHARMACEUTICALS CORPORATION
公开号:US20160095924A1
公开(公告)日:2016-04-07
The present invention provides superior compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel 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 specific target proteins 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.
本发明提供了卓越的组合物和方法,用于将治疗剂传递到细胞中。特别是,这些包括新型脂质和核酸-脂质颗粒,可有效地封装核酸并将封装的核酸高效地传递到体内的细胞中。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靶蛋白。此外,与以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。