[EN] IMPROVED AMINO LIPIDS AND METHODS FOR THE DELIVERY OF NUCLEIC ACIDS<br/>[FR] LIPIDES AMINÉS AMÉLIORÉS ET PROCÉDÉS D'ADMINISTRATION D'ACIDES NUCLÉIQUES
申请人:TEKMIRA PHARMACEUTICALS CORP
公开号:WO2010042877A1
公开(公告)日:2010-04-15
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.
本发明提供了用于将治疗剂传递给细胞的优越组合物和方法。具体来说,这些包括新型脂质和核酸-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。本发明的组合物具有高度的效力,因此可以以相对较低的剂量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在艺术领域中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。