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    Neuronal gene transfer of adenovirus and herpes virus vectors
    3.
    发明授权
    Neuronal gene transfer of adenovirus and herpes virus vectors 失效
    腺病毒和疱疹病毒载体的神经元基因转移

    公开(公告)号：US07341718B2

    公开(公告)日：2008-03-11

    申请号：US10476076

    申请日：2002-05-15

    申请人： Stéphanie Millecamps-Navarro ,  Martine Barkats ,  Jacques Mallet

    发明人： Stéphanie Millecamps-Navarro ,  Martine Barkats ,  Jacques Mallet

    IPC分类号： A61K48/00 ,  C12N15/861 ,  C12N15/869

    CPC分类号： A61K48/00 ,  A61K38/1709 ,  A61K38/185

    摘要： The present invention is related to compositions and methods for the delivery of nucleic acids to neurons in a mammal, and uses thereof. The present invention specifically discloses the use of compounds that cause synaptic nerve sprouting to increase neuron retrograde transport of a vector or a product (a polypeptide or a nucleic acid for example) in a mammal. The invention is also based on the use of a compound that interacts with synaptosomal associated proteins to increase neuron retrograde transport of a vector or a product such as one cited above in a mammal. The invention also relates to a product comprising a viral vector comprising a transgene and a compound that causes synaptic nerve sprouting, for sequential use for delivering said transgene to neurons by retrograde transport and its uses for the preparation of a composition used as a treatment in several neurological disorders. The methods and compositions of this invention can be used to deliver various transgenes, such as markers, vaccines, therapeutic genes etc., and are suitable for experimental, therapeutic or various other applications.

    摘要翻译： 本发明涉及将哺乳动物中的核酸递送给神经元的组合物和方法及其用途。 本发明具体公开了使用引起突触神经发芽的化合物来增加哺乳动物中载体或产物（例如多肽或核酸）的神经元逆行转运。 本发明还基于与突触体相关蛋白相互作用的化合物在哺乳动物中增加载体或产物（如上文引用的产物）的神经元逆行转运的用途。 本发明还涉及包含病毒载体的产品，其包含转基因和引起突触神经萌芽的化合物，用于顺序用于通过逆行转运将所述转基因递送至神经元及其用于制备用于多种治疗的组合物的用途 神经障碍。 本发明的方法和组合物可用于递送各种转基因，例如标记物，疫苗，治疗基因等，并且适用于实验，治疗或各种其它应用。

    Recombinant adenoviruses coding for glial-derived neurotrophic factor (GDNF)
    5.
    发明授权
    Recombinant adenoviruses coding for glial-derived neurotrophic factor (GDNF) 失效
    编码胶质神经营养因子（GDNF）的重组腺病毒

    公开(公告)号：US06245330B1

    公开(公告)日：2001-06-12

    申请号：US08716326

    申请日：1996-10-04

    申请人： Philippe Horellou ,  Jacques Mallet ,  Michel Perricaudet ,  Frédéric Revah ,  Emmanuelle Vigne

    发明人： Philippe Horellou ,  Jacques Mallet ,  Michel Perricaudet ,  Frédéric Revah ,  Emmanuelle Vigne

    IPC分类号： A61K4800

    CPC分类号： A61K48/00 ,  A61K38/00 ,  C07K14/475 ,  C12N15/86 ,  C12N2710/10343

    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.

    摘要翻译： 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。