申请人:Heyes James
公开号:US20130123339A1
公开(公告)日:2013-05-16
The present invention provides compositions and methods for the delivery of interfering RNAs such as siRNAs that silence APOB expression in cells such as liver cells. In particular, the nucleic acid-lipid particles provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells such as liver cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of APOB 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传递干扰RNA(如siRNA)的组合物和方法,用于在细胞中(如肝细胞)沉默APOB表达。具体来说,核酸-脂质颗粒能够有效地封装核酸,并有效地将封装的核酸传递给体内的细胞,如肝细胞。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降低APOB的表达。此外,与先前已知的技术相比,本发明的组合物和方法毒性较低,并且提供了更大的治疗指数。