发明名称 |
Gene therapy for spinal muscular atrophy |
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 viral vector containing the transgene to the spinal cord. The viral vector delivers the transgene which expresses the encoded recombinant viral gene product. The viral gene product comprises HIF1 -alpha. Also provided are compositions for delivery of a transgene product to a subject's spinal cord. |
申请公布号 |
LT2497500(T) |
申请公布日期 |
2016.12.27 |
申请号 |
LT20120157957T |
申请日期 |
2007.10.03 |
申请人 |
Genzyme Corporation |
发明人 |
O`Riordan, Catherine;Wadsworth, Samuel |
分类号 |
A61K48/00;A61P21/00;C12N15/864 |
主分类号 |
A61K48/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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