发明授权
- 专利标题： Lentiviral vectors encoding clotting factors for gene therapy
- 专利标题（中）： 编码用于基因治疗的凝血因子的慢病毒载体
-
申请号： US10145289申请日： 2002-05-14
-
公开(公告)号： US06808905B2公开(公告)日： 2004-10-26
- 发明人: James G. McArthur , Dale John Talbot , Andrew D. Simmons , Ryan McGuinness , Michael Kelly , Lisa V. Tsui , Thomas Dull
- 申请人： James G. McArthur , Dale John Talbot , Andrew D. Simmons , Ryan McGuinness , Michael Kelly , Lisa V. Tsui , Thomas Dull
- 主分类号： C12N516
- IPC分类号： C12N516
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.