Nucleic acid-lipopolymer compositions

核酸‑脂聚合物组合物

Abstract

本发明提供了增加核酸转染效率的组合物、方法和应用。在一方面,一种药物组合物可以包含与悬浮于等渗溶液中的阳离子脂聚合物缩合的浓度为至少约0.5mg/ml的核酸,其中所述阳离子脂聚合物包含具有与其主链共价连接的胆固醇和聚乙二醇的阳离子聚合物主链,并且其中胆固醇与阳离子聚合物主链的摩尔比是约0.1~约10,且聚乙二醇与阳离子聚合物主链的摩尔比是约0.1~约10。所述组合物还可以包含填料赋形剂。
Compositions, methods, and applications that increase the efficiency of nucleic acid transfection are provided. In one aspect, a pharmaceutical composition may include at least about 0.5 mg/ml concentration of a nucleic acid condensed with a cationic lipopolymer suspended in an isotonic solution, where the cationic lipopolymer includes a cationic polymer backbone having cholesterol and polyethylene glycol covalently attached thereto, and wherein the molar ratio of cholesterol to cationic polymer backbone is within a range of from about 0.1 to about 10, and the molar ratio of polyethylene glycol to cationic polymer backbone is within a range of from about 0.1 to about 10. The composition further may include a filler excipient.

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