| Project/Area Number |
23710240
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| Research Category |
Grant-in-Aid for Young Scientists (B)
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| Allocation Type | Multi-year Fund |
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| Research Field |
Applied genomics
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| Research Institution | Kyoto University |
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Principal Investigator |
WOLTJEN Knut 京都大学, 白眉センター, 准教授 (50589489)
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| Project Period (FY) |
2011 – 2012
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| Project Status |
Completed (Fiscal Year 2013)
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| Budget Amount *help |
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2012: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2011: ¥2,860,000 (Direct Cost: ¥2,200,000、Indirect Cost: ¥660,000)
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| Keywords | 遺伝子ターゲティング / ヌクレアーゼ / iPS細胞 / TALEN nuclease / transposon / cardiomyocyte / SNP / ion channel / Gene targeting / CRISPR/Cas9 nuclease / iPS Cell / long QT-syndrome / TALEヌクレアーゼ / トランスポゾン |
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| Research Abstract |
In this study, we aimed to combine TALEN (TAL-Effector Nuclease) technology and piggyBac transposon-based selection cassettes to establish novel gene-modification technology in human iPS cells. We succeeded in genetic modification using TALENs, disrupting the HPRT gene and introducing transgene cassettes into the AAVS1 locus in human iPS cells. In addition, we also succeeded in single base substitution within the HPRT gene using TALEN disruption and a single-stranded oligonucleotide repair template. Based on the gene modification technology that we have established in human iPS cells, we propose applications in gene correction of disease-specific iPS cells, or the production of disease models to understand pathogenesis and establish clinical treatments.
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