COMPOSITIONS AND METHODS FOR SILENCING APOLIPOPROTEIN B
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.
The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic 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 a specific target protein 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.
Synthesis of Pyridinium Amphiphiles Used for Transfection and Some Characteristics of Amphiphile/DNA Complex Formation
作者:Arthur A. P. Meekel、Anno Wagenaar、Jarmila Šmisterová、Jessica E. Kroeze、Peter Haadsma、Bouke Bosgraaf、Marc C. A. Stuart、Alain Brisson、Marcel H. J. Ruiters、Dick Hoekstra、Jan B. F. N. Engberts