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    • 1. 发明授权
    • Recombinant adenoviruses coding for glial-derived neurotrophic factor (GDNF)
    • 编码胶质神经营养因子(GDNF)的重组腺病毒
    • US06245330B1
    • 2001-06-12
    • US08716326
    • 1996-10-04
    • Philippe HorellouJacques MalletMichel PerricaudetFrédéric RevahEmmanuelle Vigne
    • Philippe HorellouJacques MalletMichel PerricaudetFrédéric RevahEmmanuelle Vigne
    • A61K4800
    • A61K48/00A61K38/00C07K14/475C12N15/86C12N2710/10343
    • Recombinant adenoviruses comprising a heterologous DNA sequence coding for glial-derived neurotrophic growth factor (GDNF) are provided. The recombinant adenoviruses are useful in a method of expressing GDNF in a cell, wherein the cell is present in a mammal suffering from Parkinson's disease, comprising infecting said cell with a replication-defective recombinant adenovirus comprising a DNA sequence encoding GDNF operably linked to a promoter by administering the adenovirus into cells of the central nervous system. The recombinant adenoviruses of the invention are also useful in a method of treating Parkinson's disease comprising administering into cells of the central nervous system of a mammal suffering therefrom a replication defective recombinant adenovirus comprising ITRs, an encapsidation sequence and a DNA sequence encoding GDNF operably linked to a promoter, wherein the adenovirus E1 gene is non-functional and GDNF is expressed at a level that provides a therapeutic effect.
    • 提供了包含编码神经胶质来源的神经营养生长因子(GDNF)的异源DNA序列的重组腺病毒。 所述重组腺病毒可用于在细胞中表达GDNF的方法,其中所述细胞存在于患有帕金森病的哺乳动物中,所述方法包括用复制缺陷型重组腺病毒感染所述细胞,所述复制缺陷型重组腺病毒包含与启动子可操作地连接的编码GDNF的DNA序列 通过将腺病毒施用于中枢神经系统的细胞。 本发明的重组腺病毒也可用于治疗帕金森病的方法,包括向患有其的哺乳动物的中枢神经系统的细胞施用包含ITR的复制缺陷型重组腺病毒,包封序列和编码GDNF的DNA序列,其可操作地连接到 启动子,其中腺病毒E1基因是非功能性的,并且GDNF以提供治疗效果的水平表达。