申请人:Tekmira Pharmaceuticals Corporation
公开号:US20140179761A1
公开(公告)日:2014-06-26
The present invention provides targeting lipids of structure
L
100
-linker-L
101
(CI),
where L
100
is a lipid, lipophile, alkyl, alkenyl or alkynyl, L
101
is a ligand or —CH
2
CH
2
(OCH
2
CH
2
)
p
O(CH
2
)
q
CH
2
-ligand, p is 1-1000, and q is 1-20. In addition, the invention provides 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.
本发明提供了结构为L100-连接体-L101(CI)的靶向脂质,其中L100是一种脂质、亲脂性、烷基、烯基或炔基,L101是一种配体或—CH2CH2(OCH2CH2)pO(CH2)qCH2-配体,p为1-1000,q为1-20。此外,本发明还提供了递送治疗剂到细胞的组合物和方法。特别地,这些包括新型脂质和核酸-脂质颗粒,它们提供了核酸的高效封装和封装核酸的高效递送到体内细胞的方法。