| Project/Area Number |
26461530
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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 | Kitasato University |
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Principal Investigator |
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| Co-Investigator(Renkei-kenkyūsha) |
NAGAO Kazuaki 北里大学, 医学部, 講師 (60392487)
KAMEYAMA Kohzoh 北里大学, 医学部, 講師 (40214556)
TAKAYAMA Yoshinaga 北里大学, 医学部, 講師 (90245407)
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| Project Period (FY) |
2014-04-01 – 2017-03-31
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| Project Status |
Completed (Fiscal Year 2016)
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| Budget Amount *help |
¥4,810,000 (Direct Cost: ¥3,700,000、Indirect Cost: ¥1,110,000)
Fiscal Year 2016: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2015: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2014: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
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| Keywords | 母斑基底細胞癌症候群 / CRISPR/Cas9システム / iPS細胞 / 遺伝子編集 / PTCH1 / 髄芽腫 / LOH / モザイク / CRISPR/Cas9 |
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| Outline of Final Research Achievements |
We have successfully established nevoid basal cell carcinoma syndrome (NBCCS)-specific induced pluripotent stem cells (iPS cells). Medulloblastoma was formed in all the teratomas generated from all NBCCS-specific iPSC clones established from 4 different patients. These results demonstrated that NBCCS-specific iPSCs can be a good model of NBCCS-derived medulloblastoma, and thereby used for drug screening to prevent or treat medulloblastomas.
In addition, by using CRISPR/Cas9 system, we also generated iPS cells in which mutation was introduced in remaining normal PTCH1 allele. These cells exhibited accelerated cell growth and expression of Gli target genes indicating up-regulation of hedgehog signaling. Moreover, we generated iPS cells in which mutated allele was edited and corrected.
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