| Project/Area Number |
14207033
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| Research Category |
Grant-in-Aid for Scientific Research (A)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Circulatory organs internal medicine
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| Research Institution | The University of Tokyo |
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Principal Investigator |
TOYO-OKA Teruhiko The University of Tokyo, Health Service Center, Professor, 保健センター, 教授 (00146151)
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| Co-Investigator(Kenkyū-buntansha) |
KURACHI Kotoku National Institute of Advanced Industrial Science and Technology, Age Dimension Research Center, Dean, 年齢軸生命工学研究センター, センター長(研究職) (70344223)
OZAWA Keiya Jichi Medical School, Division of Gene Therapy, Professor, 遺伝子治療部, 教授 (30137707)
TOBKUNAGA Katsushi The University of Tokyo, Graduate School of Medicine, Professor, 大学院・医学系研究科, 教授 (40163977)
UEHARA Yoshio The University of Tokyo, Health Service Center, Associate Professor, 保健センター, 助教授 (40184965)
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| Project Period (FY) |
2002 – 2004
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| Project Status |
Completed (Fiscal Year 2004)
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| Budget Amount *help |
¥50,050,000 (Direct Cost: ¥38,500,000、Indirect Cost: ¥11,550,000)
Fiscal Year 2004: ¥8,840,000 (Direct Cost: ¥6,800,000、Indirect Cost: ¥2,040,000)
Fiscal Year 2003: ¥16,250,000 (Direct Cost: ¥12,500,000、Indirect Cost: ¥3,750,000)
Fiscal Year 2002: ¥24,960,000 (Direct Cost: ¥19,200,000、Indirect Cost: ¥5,760,000)
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| Keywords | Cardiac failure / Gene therapy / Pre-clinical studies / 細胞膜の透過性 / ジストロフィン / 拡張型心筋症 / サルゴグリカン / 細胞移植 / rAAVベクター |
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| Research Abstract |
Considerable results was obtained in the first 3 objects, i.e. (1)Preparation of advanced heart failure of human-mimicking type in large animals, (2)Verification of safety and efficacy of gene therapy and (3)Development of reconstructed cell-based therapy. We have identified responsible gene for congenital dilated cardiomyopathy (DCM) as a mutation of δ-sarcoglycan gene (Sakamoto et al., PNAS,1997). Then, we have for the first time succeeded in gene therapy with using stable and long-lasting rAAV vector (Kawada et al, PNAS,2002). Different from other vectors, rAAV was safe and biologically inert. To extend our project for clinical study using apes, we had to consume one and a half year for the permission of the Ministry of Education, Culture, Science and Technology. Our final paradigm that heart failure is proceeded by a common mechanism secondary to dystrophin disruption and increment of sarcolemmal permeability, irrespective of hereditary or acquired origins and acute or chronic processes was common to heart failure in general including human DCM was extremely precious, because these data were not ethically obtained without employing apes (Toyo-oka et al., PNAS,2004). These results would provide a promising fruit to develop for the clinical application, and we do ask future fund.
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