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Cation characteristic parents crocking characteristic micellar mixture

机译:阳离子特征的父母易碎的特征胶束混合物

摘要

The effective introduction of foreign genes and other biologically active molecules into targeted mammalian cells is a challenge still facing those skilled in the art. Gene therapy, for example, requires successful transfection of target cells in a patient. The present invention relates to novel micellar complexes of cationic amphiphilic compounds that facilitate delivery of biologically active molecules to the targeted cells of a mammal. The novel micellar complexes are comprised of a cationic amphiphile, a biologically active molecule, a derivative of polyethylene glycol (PEG), and optionally, a co-lipid. A further aspect of the invention is the use of targeting absents in any of the methods that effectuate the delivery of biologically active molecules into the cells of mammals. A targeting agent is usually any molecule, peptide sequence, or large protein that preferentially targets or binds to specific mammalian cells.
机译:有效地将外源基因和其他生物活性分子引入靶向的哺乳动物细胞是本领域技术人员仍然面临的挑战。例如,基因疗法需要成功转染患者体内的靶细胞。本发明涉及阳离子两亲化合物的新型胶束复合物,其促进生物活性分子向哺乳动物靶细胞的递送。新型的胶束复合物由阳离子两亲物,生物活性分子,聚乙二醇(PEG)的衍生物和任选的共脂质组成。本发明的另一个方面是在缺乏将靶标中的生物活性分子传递到哺乳动物细胞中的任何方法中的靶向缺失的用途。靶向剂通常是优先靶向或结合特定哺乳动物细胞的任何分子,肽序列或大蛋白。

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