发明名称 |
Methods and compositions for treatment of myotonia |
摘要 |
The present invention provides methods and compositions for the treatment of diseases associated with aberrant microsatellite expansions. Methods of the present invention comprise the use of recombinant adeno-associated virus vectors containing a transgene encoding at least one muscleblind protein. The present invention also provides an animal model for a disease associated with aberrant microsatellite expansion.
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申请公布号 |
US7964570(B2) |
申请公布日期 |
2011.06.21 |
申请号 |
US20050591883 |
申请日期 |
2005.03.10 |
申请人 |
UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC. |
发明人 |
SWANSON MAURICE S.;KANADIA RAHUL N.;THORNTON CHARLES A. |
分类号 |
A61K48/00;A61K38/17;C07H21/02;C12N5/00;C12N5/02;C12N15/113;C12N15/864;C12N15/90 |
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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