公开(公告)号：WO2017062502A1

公开(公告)日：2017-04-13

申请号：PCT/US2016/055570

申请日：2016-10-05

申请人： THE REGENTS OF THE UNIVERSITY OF COLORGO, A BODY CORPORATE

发明人： ANCHORDOQUY, Thomas ,  BETKER, Jamies, L.

IPC分类号： A61K9/107 ,  A61K9/127 ,  A61K47/28 ,  A61K47/48 ,  C12N15/88

CPC分类号： C12N15/88 ,  A61K48/00

摘要： Lipoplex formulations and uses thereof. In particular, the lipoplexes include at least one naturally-occurring cationic amphiphile, at least one C18-30 saturated fatty acid, cholesterol, at least one nucleic acid, and have a low charge ratio. The lipoplexes are useful for in vivo or in vitro delivery of one or more agents (e.g., a polyanionic therapeutic or an antisense therapeutic, such as an RNAi agent) and allow prolonged expression of these agents, which may be distributed via endogenous cellular pathways to surrounding cells or tissue.

摘要翻译： Lipoplex制剂及其用途。 特别地，脂质复合物包括至少一种天然存在的阳离子两亲物，至少一种C18-30饱和脂肪酸，胆固醇，至少一种核酸，并具有低电荷比。 脂质复合物可用于体内或体外递送一种或多种药剂（例如，聚阴离子治疗剂或反义治疗剂，例如RNAi剂），并允许这些药物的延长表达，其可通过内源细胞途径分布至 周围的细胞或组织。