| 发明名称 |
Methods and compositions for use in gene therapy for treatment of hemophilia |
| 摘要 |
The invention includes a composition comprising a recombinant adeno-associated viral vector comprising at least two adeno-associated virus inverted terminal repeats, a promoter/regulatory sequence, isolated DNA encoding Factor IX and accompanying 5′and 3′untranslated regions and a transcription termination.
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| 申请公布号 |
US7998734(B2) |
申请公布日期 |
2011.08.16 |
| 申请号 |
US20070932017 |
申请日期 |
2007.10.31 |
| 申请人 |
THE CHILDREN'S HOSPITAL OF PHILADELPHIA |
发明人 |
HIGH KATHERINE A.;HERZOG ROLAND W. |
| 分类号 |
A61K48/00;C12N15/00;A61K38/43;A61K38/48;C12N7/01;C12N9/64;C12N15/86;C12N15/864 |
主分类号 |
A61K48/00 |
| 代理机构 |
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代理人 |
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