发明名称 |
Methods and Compositions for use in Gene Therapy for Treatment of Hemophilia |
摘要 |
Provided are compositions of viruses, the viruses including a recombinant adeno-associated virus vector comprising at least two adeno-associated virus inverted terminal repeats, a promoter/regulatory sequence, isolated DNA encoding a blood coagulation factor and accompanying 5′and 3′untranslated regions and a transcription termination sequence.
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申请公布号 |
US2011263690(A1) |
申请公布日期 |
2011.10.27 |
申请号 |
US201113178992 |
申请日期 |
2011.07.08 |
申请人 |
THE CHILDREN'S HOSPITAL OF PHILADELPHIA |
发明人 |
HIGH KATHERINE A.;HERZOG ROLAND W. |
分类号 |
A61K48/00;A61K38/43;A61K38/48;A61P7/04;C12N7/01;C12N9/64;C12N15/63;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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