Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
    1.
    发明授权
    Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders 有权
    用于肌萎缩性侧索硬化症和其他脊髓障碍的基因治疗

    公开(公告)号:US09034836B2

    公开(公告)日:2015-05-19

    申请号:US12328267

    申请日:2008-12-04

    摘要: This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.

    摘要翻译: 本公开提供了用于治疗影响受试者的运动功能和控制的疾病或损伤的方法和组合物。 一方面,本发明通过将包含转基因的重组神经营养病毒载体给予脑,将转基因产物递送至受试者的脊髓。 病毒载体将转基因递送到易受病毒感染并表达编码的重组病毒基因产物的脑区域。 还提供了通过将含有转基因的重组神经营养病毒载体给予受试者的脑,将转基因产物递送至受试者的脊髓的组合物。