| Budget Amount *help |
¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000)
Fiscal Year 2019: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2018: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2017: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
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| Outline of Final Research Achievements |
Induced pluripotent stem cells (iPSCs) have been established from nevoid basal cell carcinoma syndrome (NBCCS) patients (PTCH1+/- iPSCs). Teratomas that developed from these iPSCs contained medulloblastoma tissues. Interestingly, these medulloblastomas carried second hit mutations in the normal allele of the PTCH1 gene.
Furthermore, using CRISPR/Cas9 system, we established iPSCs carrying mutations in both alleles of PTCH1 (PTCH1-/- iPSCs). Teratomas that developed from PTCH1-/- iPSCs mostly consisted of ectoderm tissues including medulloblastoma. Endoderm and mesoderm tissues were rarely observed, indicating that PTCH1-/- iPSCs have a strong tendency to differentiate into ectoderm cells.
These results are expected to contribute to the understanding the molecular mechanism of tumor formation in NBCCS patients and screening candidate drugs for the treatment of these tumors.
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