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公开(公告)号:US06808905B2
公开(公告)日:2004-10-26
申请号:US10145289
申请日:2002-05-14
申请人: James G. McArthur , Dale John Talbot , Andrew D. Simmons , Ryan McGuinness , Michael Kelly , Lisa V. Tsui , Thomas Dull
发明人: James G. McArthur , Dale John Talbot , Andrew D. Simmons , Ryan McGuinness , Michael Kelly , Lisa V. Tsui , Thomas Dull
IPC分类号: C12N516
CPC分类号: C12N7/00 , A61K48/00 , C07K14/755 , C12N9/644 , C12N15/86 , C12N2740/10043 , C12N2740/16043 , C12N2740/16052 , C12N2830/008 , C12N2830/15 , C12Y304/21022
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.
摘要翻译: 公开了重组慢病毒和用于转基因递送的转移载体以及使用这种载体进行基因治疗的方法。 本发明提供第三代慢病毒包装系统和用于产生重组慢病毒的一组载体,以及用于优化肝脏特异性转基因递送的新型组织特异性增强子和启动子元件。 转基因优选为凝血因子如人因子IX(hFIX)或人因子VIII(hFVIII),可用于治疗血友病。
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公开(公告)号:US07179903B2
公开(公告)日:2007-02-20
申请号:US10885457
申请日:2004-07-06
申请人: James G. McArthur , Dale John Talbot , Andrew D. Simmons , Ryan McGuinness , Michael Kelly , Lisa V. Tsui , Thomas Dull
发明人: James G. McArthur , Dale John Talbot , Andrew D. Simmons , Ryan McGuinness , Michael Kelly , Lisa V. Tsui , Thomas Dull
IPC分类号: C07H21/04 , C12N15/11 , C12N15/67 , C12N15/85 , C12N15/867
CPC分类号: C12N7/00 , A61K48/00 , C07K14/755 , C12N9/644 , C12N15/86 , C12N2740/10043 , C12N2740/16043 , C12N2740/16052 , C12N2830/008 , C12N2830/15 , C12Y304/21022
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.
摘要翻译: 公开了重组慢病毒和用于转基因递送的转移载体以及使用这种载体进行基因治疗的方法。 本发明提供第三代慢病毒包装系统和用于产生重组慢病毒的一组载体,以及用于优化肝脏特异性转基因递送的新型组织特异性增强子和启动子元件。 转基因优选为凝血因子如人因子IX(hFIX)或人因子VIII(hFVIII),可用于治疗血友病。
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