| Project/Area Number |
22K20740
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| Research Category |
Grant-in-Aid for Research Activity Start-up
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| Allocation Type | Multi-year Fund |
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| Review Section |
0802:Biomedical structure and function and related fields
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| Research Institution | Kobe University |
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Principal Investigator |
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| Project Period (FY) |
2022-08-31 – 2024-03-31
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| Project Status |
Completed (Fiscal Year 2023)
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| Budget Amount *help |
¥2,860,000 (Direct Cost: ¥2,200,000、Indirect Cost: ¥660,000)
Fiscal Year 2023: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2022: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
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| Keywords | tissue regeneration / enteric nervous system / endogenous stem cell / Hirschsprung disease / HSCR / stem cell |
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| Outline of Research at the Start |
By deploying stem cells that already exist in the living body, the proposed study aims to assess the potential application of intrinsic enteric nervous system(ENS) stem cells for regenerating ENS in Hirschsprung disease (HSCR) as a model for a congenital stem cell disease.
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| Outline of Final Research Achievements |
Many congenital diseases display incomplete organ development due to impaired function of stem cells caused by toxic effects of gene mutations. While surgery is a common treatment, their effects remain unsatisfactory. Advances in genome editing offer hope for correcting these mutations in stem cells, potentially aiding organogenesis. We used Hirschsprung disease as a model for congenital diseases, a condition where intestinal nerve cells are absent, often requiring surgery. To investigate if endogenous stem cells that reside within the affected body can restore themselves, we generated a mouse model with the disease in which the causative mutation can be conditionally removed or corrected. Correcting these stem cells resulted in increased cell proliferation and ameliorate the disease severity and also enhancing the gastrointestinal motility function. This research sheds light on potential utilization of endogenous stem cells for other congenital disorders.
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| Academic Significance and Societal Importance of the Research Achievements |
科学的には、私たちの研究は先天性疾患に影響を受ける組織を再生するための内因性幹細胞の潜在能力を明らかにしました。私たちは、Hirschsprung病の画期的なマウスモデルを開発し、標的変異修正を実証しました。社会的には、これにより先天性疾患に対する治療成果の向上が期待され、特に乳幼児におけるアガングリオノーシスなどの状態の予防が進んだ幹細胞療法が提供されます。
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