AAV vector-mediated cell and gene therapy for neuronal diseases
| Project/Area Number |
22390284
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Cerebral neurosurgery
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| Research Institution | National Center of Neurology and Psychiatry |
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Principal Investigator |
OKADA Takashi 日本医科大学, 医学(系)研究科(研究院), 教授 (00326828)
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| Co-Investigator(Kenkyū-buntansha) |
KINOH Hiromi 日本医科大学, 大学院医学研究科 (60532728)
OKADA Hironori 日本医科大学, 大学院医学研究科 (80416271)
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| Project Period (FY) |
2010-04-01 – 2015-03-31
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| Project Status |
Completed (Fiscal Year 2014)
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| Budget Amount *help |
¥14,950,000 (Direct Cost: ¥11,500,000、Indirect Cost: ¥3,450,000)
Fiscal Year 2013: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2012: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2011: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2010: ¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000)
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| Keywords | 神経疾患 / 遺伝子治療 / AAVベクター / 脳神経疾患 / ウイルス / 遺伝子 |
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| Outline of Final Research Achievements |
To realize novel protein supplementation therapy for neuronal diseases, we developed an efficient AAV vector production system and vector-producing tumor-targeting cells. By using effective ion-exchange adsorbers, improved GMP production protocol for highly purified vector particles was developed. In addition to the dual ion-exchange procedures, gel filtration chromatography realized ultracentrifugation-free vector purification. As an application for the AAV-mediated protein supplementation therapy, successful transduction of the various disease models was demonstrated. Furthermore, vector-producing tumor-directed stem cells were developed to amplify therapeutic genes.
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Report
(5 results)
Research Products
(52 results)
