| Project/Area Number |
20591233
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Jikei University School of Medicine |
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Principal Investigator |
KOBAYASHI Hiroshi Jikei University School of Medicine, 医学部, 講師 (90266619)
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| Co-Investigator(Kenkyū-buntansha) |
KIMURA Takahiro 東京慈恵会医科大学, 医学部, 助教 (00307430)
KAWAI Toshinao 東京慈恵会医科大学, 医学部, 助教 (20328305)
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| Project Period (FY) |
2008 – 2010
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| Project Status |
Completed (Fiscal Year 2010)
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| Budget Amount *help |
¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000)
Fiscal Year 2010: ¥650,000 (Direct Cost: ¥500,000、Indirect Cost: ¥150,000)
Fiscal Year 2009: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2008: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
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| Keywords | 遺伝 / 先天異常学 / 遺伝子治療 / Krabbe病 / レンチウイルスベクター / レンチウイルス / クラッベ病 / GALC / GFP |
|---|
| Research Abstract |
We investigated the effect of lenti-virus mediated gene therapy for Krabbe disease. We succeeded in preparing high titer lenti-virus including GALC, deficient enzyme in Krabbe disease expressing gene by large scale harvesting and ultracentrifuge, and injected for neonatal twitcher mouse, model for Krabbe disease resulted in efficient improvement in nutrition and life span. And we had more efficient improvement in the phenotype of twitcher mice by the combination of gene therapy and substrate reduction therapy using L-cycloserine.
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