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— (O )pO(
CH2)q -
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。