The instant invention provides for 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. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
本发明提供了一种新型阳离子脂质,可以与其他脂质成分(如
胆固醇和P
EG脂质)结合使用,形成含寡核苷酸的脂质纳米粒子。本发明的目的是提供一种阳离子脂质支架,具有增强的功效和较低的肝毒性,因为肝脏中脂质
水平较低。本发明采用低分子量的阳离子脂质,具有一个短脂链,以提高体内siRNA的输送效率和耐受性。