发明名称 |
Lentiviral vectors encoding clotting factors for gene therapy |
摘要 |
Recombinant lentiviruses and transfer vectors for transgene delivery as well as methods for gene therapy using such vectors are disclosed. The invention provides a third generation lentiviral packaging system and a set of vectors for producing recombinant lentiviruses, as well as novel tissue specific enhancer and promoter elements useful for optimizing liver specific transgene delivery. The transgene is preferably a blood clotting factor such as human factor IX (hFIX) or human factor VIII (hFVIII) and can be used for treatment of hemophilia.
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申请公布号 |
US2003077812(A1) |
申请公布日期 |
2003.04.24 |
申请号 |
US20020145289 |
申请日期 |
2002.05.14 |
申请人 |
MCARTHUR JAMES G.;TALBOT DALE JOHN;SIMMONS ANDREW D.;MCGUINNESS RYAN;KELLY MICHAEL;TSUI LISA V.;DULL THOMAS |
发明人 |
MCARTHUR JAMES G.;TALBOT DALE JOHN;SIMMONS ANDREW D.;MCGUINNESS RYAN;KELLY MICHAEL;TSUI LISA V.;DULL THOMAS |
分类号 |
A61K48/00;C07K14/755;C12N5/08;C12N5/10;C12N9/64;C12N15/12;C12N15/867;(IPC1-7):C12N7/00 |
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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