2015 Fiscal Year Final Research Report
Development of gene therapy approaches towards hemophilia A
Project/Area Number |
25461460
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Research Category |
Grant-in-Aid for Scientific Research (C)
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Allocation Type | Multi-year Fund |
Section | 一般 |
Research Field |
Hematology
|
Research Institution | Jichi Medical University |
Principal Investigator |
|
Project Period (FY) |
2013-04-01 – 2016-03-31
|
Keywords | AAVベクター / 血友病 / 遺伝子治療 |
Outline of Final Research Achievements |
We tested the efficacy of factor VIII expression using AAV vectors to achieve gene therapy for hemophilia A. As the length of factor VIII gene exceeds the packaging capacity of AAV, we constructed vectors using essential parts of factor VIII gene, combined with short, liver-specific promoter. Expression of factor VIII were achieved both in vitro and in vivo (mouse) experiments. On the other hand, the efficiency of vector production was much lower than the regular sized vectors. Further investigation is required to optimize the vector structure, mainly testing shorter promoter elements to improve the vector production rate.
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Free Research Field |
遺伝子治療
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