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