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Recombinant adenoviruses coding for glial-derived neurotrophic factor (GDNF)

机译:编码胶质细胞源性神经营养因子(GDNF)的重组腺病毒

摘要

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.
机译:提供了重组腺病毒,其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。所述重组腺病毒可用于在细胞中表达GDNF的方法中,其中所述细胞存在于患有帕金森氏病的哺乳动物中,包括用复制缺陷型重组腺病毒感染所述细胞,所述重组腺病毒包含与启动子有效连接的编码GDNF的DNA序列。通过将腺病毒施用于中枢神经系统细胞。本发明的重组腺病毒还可用于治疗帕金森氏病的方法,该方法包括向患有该疾病的哺乳动物的中枢神经系统的细胞中施用复制缺陷型重组腺病毒,该重组腺病毒包含ITR,衣壳化序列和编码GDNF的DNA序列,该序列可操作地连接至一种启动子,其中腺病毒E1基因是无功能的,并且GDNF以提供治疗效果的水平表达。

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