Establishment of the next generation treatment for hemophilia A by autologous cell implantation
| Project/Area Number |
25293237
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Partial Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Nara Medical University |
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Principal Investigator |
Shima Modori 奈良県立医科大学, 医学部, 教授 (30162663)
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| Co-Investigator(Kenkyū-buntansha) |
MATSUI Hideto 奈良県立医科大学, 医学部, 講師 (00571027)
HOTTA Akitsu 京都大学, iPS細胞研究所, 特定拠点助教 (50578002)
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| Project Period (FY) |
2013-04-01 – 2016-03-31
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| Project Status |
Completed (Fiscal Year 2015)
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| 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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| Keywords | hemophilia A / gene therapy / piggyBack / transposon / factor Ⅷ / 血友病Ａ / 遺伝子治療 / 細胞治療 / 血友病A / 自己細胞 / 細胞移植 / 免疫応答 |
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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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Report
(4 results)
Research Products
(21 results)
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[Presentation] Novel gene therapy strategy for hemophilia A by hydrodynamic gene delivery combined with non-viral piggyBac transposon vector in canine model2015
Author(s)
Noda M, Matsui H, Matsunari Y, Ochi S, Shima M, Fukuoka Y, Sato T, Anai H, Kichikawa K, Nakayama M, Hotta A, Sugimoto M.
Organizer
2015 Annual Meeting of the American Society of Hematology
Place of Presentation
Orlando, Florida, USA
Year and Date
2015-12-07
Related Report
Int'l Joint Research
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