| Project/Area Number |
26670264
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Applied pharmacology
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| Research Institution | University of Tsukuba |
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Principal Investigator |
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| Co-Investigator(Kenkyū-buntansha) |
三輪 佳宏 筑波大学, 医学医療系, 講師 (70263845)
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| Co-Investigator(Renkei-kenkyūsha) |
Nakanishi Mahito 産業技術総合研究所, 創薬基盤研究部門, ヒト細胞医工学研究ラボ長 (10172355)
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| Research Collaborator |
Ito Masashi エーザイ株式会社
Aso Masayuki セルジェンテック株式会社
Hasegawa Yuichi 筑波大学, 医学医療系, 准教授
Oneda Osamu 筑波大学, 医学医療系, 教授
Nishimura Ken 筑波大学, 医学医療系, 助教
Hisatake Koji 筑波大学, 医学医療系, 教授
Hashimoito Koichi 筑波大学, 医学医療系, 教授
Yanagi Kennichi 筑波大学, 医学医療系, 教授
Chiba Shigeru 筑波大学, 医学医療系, 教授
Sakasai Tomoki 筑波大学, 人間総合科学研究科
Tanaka Junko 筑波大学, 医学医療系, 研究員
Yamaki Yuni 筑波大学, 附属病院, 講師
Imagawa Kazuo 筑波大学, 附属病院, 助教
Fukushima Hiroko 筑波大学, 医学医療系, 講師
Fujiyama Satoshi 筑波大学, 人間総合科学研究科
Noguchi Emiko 筑波大学, 医学医療系, 教授
Sumazaki Ryo 筑波大学, 客員教授
Isoyama Shigemi 筑波大学, 医学医療系技術室
Mishima Hajime 筑波大学, 医学医療系, 准教授
Fujisawa Chizuko 筑波大学, つくば臨床医学研究開発機構
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| Project Period (FY) |
2014-04-01 – 2017-03-31
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| Project Status |
Completed (Fiscal Year 2016)
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| Budget Amount *help |
¥3,770,000 (Direct Cost: ¥2,900,000、Indirect Cost: ¥870,000)
Fiscal Year 2016: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2015: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2014: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
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| Keywords | 遺伝子治療 / 血友病Ａ / 全長型 / 全長型第VIII血液凝固因子 / 血友病 / RNAベクター / 脂肪細胞 / 生体イメージング / ＲＮＡベクター |
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| Outline of Final Research Achievements |
The following results have been shown by this research
(1)A novel RNA vector can carry genes up to 13.5 kb altogether. We detected an activity of coagulation factor VIII (FVIII) in the culture spernatant of transfected cells. Concentration of FVIII was the same between a culture of full length (FL) FVIII secreting cells and B domain deleted (BDD) FVIII secreting cells.(2)We observed a therapeutic effect by transplantation of mouse FVIII secreting cells to hemophilia A mice.(3)Only local distribution of RNA vector was confirmed after intramuscular injection, subcutaneous injection, intraperitoneal injection or enema by both in vivo and ex vivo gene therapy, under non-invasive bio-imaging. We used iRFP gene carrying RNA vector to observe distribution or migration of gene transduced cells using a fluorescent detector.
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