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    Monocytes as a gene delivery vector for secreted proteins to treat Alzheimer'S disease
    18.
    发明授权
    Monocytes as a gene delivery vector for secreted proteins to treat Alzheimer'S disease 失效
    单核细胞作为分泌蛋白质治疗阿尔茨海默氏病的基因递送载体

    公开(公告)号：US08518391B1

    公开(公告)日：2013-08-27

    申请号：US12610815

    申请日：2009-11-02

    申请人： Dave Morgan ,  Siddharth G. Kamath ,  Lori Lebson

    发明人： Dave Morgan ,  Siddharth G. Kamath ,  Lori Lebson

    IPC分类号： A61K48/00 ,  C12N5/078

    CPC分类号： C12N5/0645 ,  A61K48/0075 ,  C07K2319/02 ,  C12N9/6494 ,  C12N2510/00

    摘要： Disclosed is a method for the treatment of a neurodegenerative disorder by isolating a plurality of monocytic cells and introducing a vector containing a gene that expresses a protease capable of degrading amyloid peptide into the isolated monocytic cells. The modified cells are then administered to the patient. Preferably the introduced gene is selected from the group consisting of neprilysin, insulin degrading enzyme and endothelin converting enzyme. The protease capable of degrading amyloid peptide is secreted from the monocytic cells into the extracellular space thereof. According to one variant, the gene that expresses a protease capable of degrading amyloid peptide is the NEP gene with a deletion in the membrane binding domain and/or an appended signal peptide to drive secretion of the modified gene product. This invention contemplates the use of heterologous and autologos (the monocytic cells are obtained from the patient) transplants.

    摘要翻译： 公开了一种通过分离多个单核细胞并将含有表达能够将淀粉样蛋白肽降解的蛋白酶的基因的载体引入到分离的单核细胞中的方法来治疗神经变性疾病。 然后将修饰的细胞施用于患者。 优选地，引入的基因选自由尿丙氨酸，胰岛素降解酶和内皮素转化酶组成的组。 能够降解淀粉样蛋白肽的蛋白酶从单核细胞分泌到其细胞外空间中。 根据一个变体，表达能够降解淀粉样蛋白肽的蛋白酶的基因是在膜结合域中具有缺失的NEP基因和/或附加的信号肽以驱动修饰的基因产物的分泌。 本发明考虑使用异源和自体（单核细胞获自患者）移植物。