申请人:Hope Michael J.
公开号:US09139554B2
公开(公告)日:2015-09-22
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.
本发明提供了优越的组合物和方法,用于向细胞提供治疗剂。特别地,这些组合物包括新型的脂质和核酸-脂质颗粒,能够高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。本发明的组合物具有高度的效力,因此可在相对较低的剂量下有效地靶向特定的蛋白质。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低,提供了更大的治疗指数。