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 signal, and methods of use thereof.
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申请公布号 |
US6093392(A) |
申请公布日期 |
2000.07.25 |
申请号 |
US19980038910 |
申请日期 |
1998.03.12 |
申请人 |
CHILDRENS HOSPITAL OF PHILDELPHIA |
发明人 |
HIGH, KATHERINE A.;HERZOG, ROLAND W. |
分类号 |
A61K48/00;A61K38/43;A61K38/48;C12N7/01;C12N9/64;C12N15/864;(IPC1-7):A61K48/00;C12N15/86 |
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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