2002 Fiscal Year Final Research Report Summary
Hemophilia A gene therapy using Adenovirus vector (Mouse model)
| Project/Area Number |
12670772
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Department of Pediatrics, Nara Medical University |
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Principal Investigator |
NAKA Hiroyuki Nara Medical University, Department of Pediatrics, Research Associate, 小児科学教室, 助手 (40281761)
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| Project Period (FY) |
2000 – 2002
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| Keywords | Hemophilia A / Gene Therapy / Adenovirus / Lentivirus |
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| Research Abstract |
We used a first generation adenovirus vector, in which El and E3 genes were deleted and inserted canine FVIII cDNA deleted B-domain, and the third generation of lentivirus vector, to assess whether functional canine FVIII could be expressed and detected in vitro and in vivo. In the case of the adenovirus vector, the biological active canine FVIII was detected both in vitro and in vivo, but the expression of in vivo study was relatively transient due to allo-antibody against expressed FVIII. In the case of Lentivirus vector, the biological active canine FVIII was also detected in vitro. In vivo study suggested the intra-portal injection was the best way for gene delivery to liver. We detected the biological FVIII antigen and activity for 8 weeks after injection. FVIII antibody was not detected at endpoint of this study.
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