Identification of genomic changes and their therapeutic effects in neuronally-differentiated induced pluripotent stem cells of autistic patients
| Project/Area Number |
25670473
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Pediatrics
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| Research Institution | University of Yamanashi |
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Principal Investigator |
KUBOTA Takeo 山梨大学, 総合研究部, 教授 (70293511)
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| Co-Investigator(Kenkyū-buntansha) |
TOYODA Atsushi 国立遺伝学研究所, 生物遺伝資源情報総合センター, 特任准教授 (10267495)
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| Project Period (FY) |
2013-04-01 – 2015-03-31
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| Project Status |
Completed (Fiscal Year 2014)
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| Budget Amount *help |
¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2014: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2013: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
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| Keywords | エピジェネティクス / エピゲノム / 治療薬 / iPS細胞 / レット症候群 / 自閉症 / 遺伝子発現制御 / バルプロ酸ナトリウム / 遺伝子発現 / てんかん |
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| Outline of Final Research Achievements |
We investigated abnormalities in the neuronal cells differentiated from the induced pluripotent stem cells (iPSC) derived from Rett syndrome patients, a representative autistic disorder caused by MECP2 mutations. As a result, we found abnormal expression of astrocyte-specific genes. Furthermore, we found that the chemicals, target to HDAC and Sin3 which are parts of MeCP2 protein complex, up-regulated Rett syndrome- and autism-associated genes. These findings suggest that malfunction of MeCP2-induced genomic changes may underlie the alteration of astrocyte-specific genes, and indicate that the chamicals examined possibly recover the gene expression which were down-regulated in Rett and autistic patients.
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Report
(3 results)
Research Products
(26 results)
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[Journal Article] Differentiation of multipotent neural stem cells derived from Rett syndrome patients is biased toward the astrocytic lineage.2015
Author(s)
Andoh-Noda T, Akamatsu W, Miyake K, MatsumotoT, Yamaguchi R, Sanosaka T, Okada Y, Kobayashi T, Ohyama M, Nakashima K, Kurosawa H, Kubota T, Okano H.
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Journal Title
Related Report
Peer Reviewed / Acknowledgement Compliant
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[Journal Article] Comparison of genomic and epigenomic expression in monozygotic twins discordant for Rett syndrome2013
Author(s)
Miyake K, Yang C, Minakuchi Y, Ohori K, Soutome M, Hirasawa T, Kazuki Y, Adachi N, Suzuki S, Itoh M, Goto Y, Andoh T, Kurosawa H, Akamatsu W, Oyama M, Okano H, Oshimura M, Sasaki M, Toyoda A, Kubota T
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Journal Title
PLoS ONE
Volume: 8
Issue: 6
Pages: e66729-e66729
DOI
Related Report
Peer Reviewed
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