| Project/Area Number |
15K15393
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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 |
Pediatrics
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| Research Institution | Fukuoka Children’s Hospital (2017)
Kyushu University (2015-2016) |
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Principal Investigator |
HARA Toshiro 地方独立行政法人福岡市立病院機構福岡市立こども病院(臨床研究部), 臨床研究部, 院長 (40150445)
|
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| Co-Investigator(Kenkyū-buntansha) |
高田 英俊 九州大学, 医学研究院, 教授 (70294931)
山元 裕之 九州大学, 環境発達医学研究センター, 特任助教 (00710170)
|
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| Project Period (FY) |
2015-04-01 – 2018-03-31
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| Project Status |
Completed (Fiscal Year 2017)
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| Budget Amount *help |
¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2016: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2015: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
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| Keywords | 遺伝子治療 / 人工ヌクレアーゼ |
|---|
| Outline of Final Research Achievements |
To examine the possibility of using gene therapy for primary immunodeficiencies, we constructed a helper-dependent adenovirus/adeno-associated virus hybrid vector to serve as a donor template, and a sequence-specific gRNA for double strand breaks. Human cord blood CD34 positive cells were infected with the hybrid vector and electroporated with gRNA and Cas9 protein. This delivery method can be a new gene-repair therapy for clinical application.
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