Muthiah Manoharan,Kallanthottathil G. Rajeev,Muthusamy Jayaraman,Akin Akinc,Shigeo Matsuda,Martin Maier
申请号：
US14362848
公开号：
US20140308304A1
申请日:
2012.12.07
申请国别(地区):
US
年份:
2014
代理人:
摘要：
The present invention relates to novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides, to facilitate the cellular uptake and endosomal escape, and to knockdown target mRNA both in vitro and in vivo. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
