| Budget Amount *help |
¥18,070,000 (Direct Cost: ¥13,900,000、Indirect Cost: ¥4,170,000)
Fiscal Year 2015: ¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000)
Fiscal Year 2014: ¥7,800,000 (Direct Cost: ¥6,000,000、Indirect Cost: ¥1,800,000)
Fiscal Year 2013: ¥5,590,000 (Direct Cost: ¥4,300,000、Indirect Cost: ¥1,290,000)
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| Outline of Final Research Achievements |
In the current gene and cell-based therapy, there are concerns over the safety of the systemic delivery of conventional viral vectors such as adverse immunological reactions or virus-mediated cytotoxicity. We established non-viral hydrodynamic gene delivery (HGD) using piggyBack transposon vector. We tested the HGD in hemophilia A mouse model with the combination of non-viral piggyBack transposon vectors which can transfer the full-length FVIII transgene. As a result, we confirmed the sustained FVIII expression for over 300 days without any immune responses. Nextly, we focused on an assessment of the safety of liver-target HGD of the piggyBack transposon vector in dogs. Liver-targeted HGD was performed using piggyBack transposon vector expressing either GFP or FVIII. By histological examination of liver samples, approximately 70-80% of hepatocytes in all 4 lobes were positive with GFP 120 days after HGD.
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