Application of human pluripotent stem cell-derived neural crest cells for cell therapy in mini-gut
| Project/Area Number |
18K08666
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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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| Review Section |
Basic Section 55020:Digestive surgery-related
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| Research Institution | National Center for Child Health and Development |
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Principal Investigator |
Sugawara Tohru 国立研究開発法人国立成育医療研究センター, 細胞医療研究部, 上級研究員 (70553460)
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| Project Period (FY) |
2018-04-01 – 2021-03-31
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| Project Status |
Completed (Fiscal Year 2020)
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| Budget Amount *help |
¥4,420,000 (Direct Cost: ¥3,400,000、Indirect Cost: ¥1,020,000)
Fiscal Year 2020: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2019: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2018: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
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| Keywords | 神経堤細胞 / 神経堤症 / ヒト多能性幹細胞 / microRNA / 小腸オルガノイド / Hirschsprung病 / RET遺伝子 / オルガノイド / ゲノム編集 / hsa-miR-302 / CRISPR/Cas9 / ヒト人工多能性幹細胞 / ヒト胚性幹細胞 / 疾患モデル / ミニ腸 / 病態モデル |
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| Outline of Final Research Achievements |
The neural crest cells (NCCs) originate at the dorsalmost region of the neural tube during the embryonic development of vertebrates. The dysfunction of NCCs is called neurocristopathy. It causes various diseases such as cardiac and intestinal dysfunction, developmental disorders, and congenital malformations as well as tumors.
In this study, we aimed to produce human pluripotent stem cell (hPSC)-derived NCCs as materials for cell therapy against neurocristopathy. In addition, we will generate hPSC-derived three-dimensional small intestinal organoid disease modelings and evaluate the function of hPSC-derived NCs.
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| Academic Significance and Societal Importance of the Research Achievements |
Hirschsprung病も神経堤症の一つで、腸管の動きを制御する腸の神経節細胞の形成不全により、生後数日の間に機能性の腸閉塞症状がみられる。また、Hirschsprung類縁疾患は同様の症状を示すが発症原因は不明であり、治療方法も確立されていない。機能的な三次元小腸オルガノイドを作製することに成功したのは今のところ世界中で本研究室のみであり、細胞治療の原材料としての神経堤細胞の評価を生体内に近い状況おこなうことでヒトに対する細胞治療の実現を加速させることが期待される点で大きな意義のある研究となる。
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Report
(4 results)
Research Products
(6 results)
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[Journal Article] Identification and therapeutic rescue of autophagosome and glutamate receptor defects in C9ORF72 and sporadic ALS neurons.2019
Author(s)
Shi Y, Hung ST, Rocha G, Lin S, Linares GR, Staats KA, Seah C, Wang Y, Chickering M, Lai J, Sugawara T, Sagare AP, Zlokovic BV, Ichida JK.
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Journal Title
JCI Insight
Volume: 4
Issue: 15
DOI
Related Report
Peer Reviewed / Open Access / Int'l Joint Research
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[Journal Article] Zscan5b Deficiency Impairs DNA Damage Response and Causes Chromosomal Aberrations during Mitosis.2019
Author(s)
Ogawa S, Yamada M, Nakamura A, Sugawara T, Nakamura A, Miyajima S, Harada Y, Ooka R, Okawa R, Miyauchi J, Tsumura H, Yoshimura Y, Miyado K, Akutsu H, Tanaka M, Umezawa A, Hamatani T.
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Journal Title
Stem Cell Reports
Volume: 12
Issue: 6
Pages: 1366-1379
DOI
Related Report
Peer Reviewed / Open Access / Int'l Joint Research
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