Brain organoids as pathological model for Dravet syndrome applicable to drug discovery research
Project/Area Number |
18K15735
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Research Category |
Grant-in-Aid for Early-Career Scientists
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Allocation Type | Multi-year Fund |
Review Section |
Basic Section 52050:Embryonic medicine and pediatrics-related
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Research Institution | Fukuoka University |
Principal Investigator |
TANAKA Yasuyoshi 福岡大学, てんかん分子病態研究所, ポスト・ドクター (50714466)
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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 |
¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2020: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2019: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2018: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
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Keywords | Dravet症候群 / 発達性てんかん性脳症 / SCN1A / Nav1.1 / 疾患特異的iPS細胞 / 脳オルガノイド / 病態モデル / GABA作動性神経細胞 / てんかん / iPS細胞 / 疾患モデル / 終脳オルガノイド / ex vivo病態モデル |
Outline of Final Research Achievements |
In this study, brain organoids in the medial ganglionic eminence (MGE) region containing neural stem cells positive for FOXG1 and NKX2.1 expression were developed from iPS cells derived from Dravet syndrome (DS) patients. The expression of parvalbumin (PV) positive inhibitory neurons was confirmed in brain organoids resembling in medial ganglionic eminence (MGE). It was also confirmed that these PV positive cells were positive for Nav1.1 expression.
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Academic Significance and Societal Importance of the Research Achievements |
てんかん等の脳神経機能異常を示す病態研究において、ヒト脳組織は利用できない。代わりにモデル動物を用いたin vivo研究が行われているが、必ずしもヒトの複雑な病態を反映しているとは言い難い。 本研究において、疾患特異的iPS細胞よりDSの病態と関連性のあるMGEOsの作製に成功した。これにより、患者の脳組織を模倣したex vivo病態モデルとして、病態の解明や新たな治療法の開発に向けた、今後のてんかん研究への応用が期待される。
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Report
(4 results)
Research Products
(8 results)