2014 Fiscal Year Final Research Report
Gene Therapy for the Krabbe disease using zinc finger nuclease system
| Project/Area Number |
24591527
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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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 |
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| Co-Investigator(Kenkyū-buntansha) |
YOTA Shimada 東京慈恵会医科大学, 総合医科学研究センター遺伝子治療研究部, 助教 (20560824)
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| Project Period (FY) |
2012-04-01 – 2015-03-31
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| Keywords | Krabbe病 / Zinc Finger / GALC |
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| Outline of Final Research Achievements |
We prepared the specific donor plasmid (DP) and zinc finger nuclease (ZFN) for homologus recombination of the point mutation (chromosome 12, exon 10) in the model mouse of Krabbe disease, and co-transfected them into the Schwann cell line origin from the model mouse by the nucleofecta (electropolation system), resulted in normalize of the point mutation, efficient high expression of GALC enzyme (deficient in the Krabbe disease) activity, reduction of pscichosine in the treated cell, and it has been maintained after some passages.
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| Free Research Field |
先天代謝異常
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