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PHARMACEUTICAL COMPOSITION IN THE FORM OF A NUCLEIC ACID LIPID COMPLEX, THE PRODUCTION THEREOF AND ITS USE IN GENE THERAPY

机译:核酸脂质复合物形式的药物组合物,其生产及其在基因治疗中的用途

摘要

The invention relates to a pharmaceutical composition in the form of a nucleic acid lipid complex containing at least one cationic lipid, at least one non-cationic lipid, at least one nucleic acid that codes for a protein used for treating vascular diseases, especially a protein having vasodilatory and/or angiogenic properties, and optionally containing appropriate auxiliary and/or addition agents. According to the invention, the cationic lipid (KL) contains a group derived from cholesterol on which at least one cationic amino group selected from a primary, secondary, tertiary amino group and/or from a quaternary ammonium salt is bound via a connecting group selected from carboxamides and carbamoyls, and via a spacer comprised of a linear or branched alkyl group with 1 to 20 carbon atoms. In addition, the size of the nucleic acid lipid complexes ranges from approximately 300 to 800 nm. The invention also relates to the production of the pharmaceutical composition and to its use in gene therapy.
机译:本发明涉及一种核酸脂质复合物形式的药物组合物,其包含至少一种阳离子脂质,至少一种非阳离子脂质,至少一种编码用于治疗血管疾病的蛋白质,特别是蛋白质的核酸具有血管扩张和/或血管生成特性,并任选地包含适当的辅助和/或添加剂。根据本发明,阳离子脂质(KL)包含衍生自胆固醇的基团,通过选自以下的连接基团结合至少一个选自伯,仲,叔氨基和/或季铵盐的阳离子氨基。衍生自羧酰胺和氨基甲酸酯,并通过由具有1至20个碳原子的直链或支链烷基组成的间隔基。另外,核酸脂质复合物的大小在约300至800nm的范围内。本发明还涉及药物组合物的生产及其在基因治疗中的用途。

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