首页> 外国专利> Cationic virosomes having a positively charged lipid bilayer membrane with an integrated viral fusogenic peptide as genetic transfer systems for use in gene therapy

Cationic virosomes having a positively charged lipid bilayer membrane with an integrated viral fusogenic peptide as genetic transfer systems for use in gene therapy

机译:阳离子病毒体,具有带正电荷的脂质双层膜和整合的病毒融合肽作为基因转移系统,用于基因治疗

摘要

A lipid vesicle with a positively charged lipid bilayer membrane containing cationic and/or polycationic lipids. At least one active viral fusogenic peptide is integrated, or covalently linked to the membrane and is capable of inducing a fusion reaction between the vesicle membrane and a lipid membrane of the target cell. The membrane comprises 10% or less by weight of phosphatidylethanolamine, and 90% or more by weight of other cationic and /or polycationic lipids, preferably phosphatidylcholine. The vesicle, in addition to containing the desired genetic material for delivery to target cells, also contains a cell-specific marker selected from the group comprising an antibody or fragment thereof, a cytokine and a growth factor. Also disclosed is the process for the manufacture of the lipid bilayer vesicle, and the use of the positively charged virosome as a medicament for carrying out non-infectious gene therapy, wherein gene therapy includes the reduction or proliferative activity of cancer, leukemic and virally infected cells.
机译:具有带阳离子和/或聚阳离子脂质的带正电的脂质双层膜的脂质囊泡。至少一种活性病毒融合肽被整合或共价连接至膜,并且能够诱导囊泡膜与靶细胞的脂质膜之间的融合反应。该膜包含按重量计10%或更少的磷脂酰乙醇胺,和按重量计90%或更多的其他阳离子和/或聚阳离子脂质,优选磷脂酰胆碱。囊泡除了含有递送至靶细胞的所需遗传物质外,还含有选自包括抗体或其片段,细胞因子和生长因子的组的细胞特异性标记。还公开了脂质双层囊泡的制造方法,以及带正电荷的病毒体作为用于进行非感染性基因治疗的药物的用途,其中基因治疗包括癌症,白血病和病毒感染的减少或增殖活性细胞。

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