METHODS AND COMPOSITIONS FOR TREATING BRAIN DISEASES
    16.
    发明申请
    METHODS AND COMPOSITIONS FOR TREATING BRAIN DISEASES 有权
    治疗脑疾病的方法和组合物

    公开(公告)号:US20140088179A1

    公开(公告)日:2014-03-27

    申请号:US14008994

    申请日:2012-04-02

    IPC分类号: A61K48/00

    摘要: The present disclosure provides methods of treating a disease in a non-rodent mammal comprising administering to the cerebrospinal fluid (CSF) of the non-rodent mammal an rAAV2 particle containing a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner effective to infect an ependymal cell in the non-rodent mammal, wherein the ependymal cell secretes the therapeutic protein so as to treat the disease.

    摘要翻译: 本公开提供了治疗非啮齿动物哺乳动物疾病的方法,包括对非啮齿动物哺乳动物的脑脊液(CSF)施用含有载体​​的rAAV2颗粒,所述载体包含插入在一对AAV之间的治疗性蛋白质的核酸 以有效感染非啮齿动物哺乳动物中的室管膜细胞的方式重复反向末端,其中室管膜细胞分泌治疗性蛋白质以治疗疾病。