申请人:Heyes James
公开号:US08865675B2
公开(公告)日:2014-10-21
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.
本发明提供了一种组合物和方法,用于递送干扰RNA(如siRNA)以在细胞中(如肝细胞)沉默APOB表达。特别地,核酸脂质颗粒提供了高效的核酸封装和有效的封装核酸递送到体内的细胞(如肝细胞)。本发明的组合物具有高度的效力,因此可以在相对较低的剂量下有效地抑制APOB。此外,本发明的组合物和方法比先前已知的组合物和方法更少毒性,提供更大的治疗指数。