Development epigenomic restoration therapy for autistic disorders
Project/Area Number |
23390272
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Research Category |
Grant-in-Aid for Scientific Research (B)
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Allocation Type | Single-year Grants |
Section | 一般 |
Research Field |
Pediatrics
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Research Institution | University of Yamanashi |
Principal Investigator |
KUBOTA Takeo 山梨大学, 医学工学総合研究部, 教授 (70293511)
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Co-Investigator(Kenkyū-buntansha) |
KUROSAWA Hiroshi 山梨大学, 医学工学総合研究部, 教授 (10225295)
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Co-Investigator(Renkei-kenkyūsha) |
OKANO Hideyuki 慶應義塾大学, 医学部, 教授 (60160694)
NAGASE Hiroki 千葉県がんセンター(研究所), 研究所長 (90322073)
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Project Period (FY) |
2011-04-01 – 2014-03-31
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Project Status |
Completed (Fiscal Year 2013)
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Budget Amount *help |
¥18,330,000 (Direct Cost: ¥14,100,000、Indirect Cost: ¥4,230,000)
Fiscal Year 2013: ¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000)
Fiscal Year 2012: ¥5,590,000 (Direct Cost: ¥4,300,000、Indirect Cost: ¥1,290,000)
Fiscal Year 2011: ¥8,060,000 (Direct Cost: ¥6,200,000、Indirect Cost: ¥1,860,000)
|
Keywords | エピジェネティクス / エピゲノム / レット症候群 / MeCP2 / 遺伝子発現 / 修復治療 / 化合物 / バルプロ酸ナトリウム / 自閉症 / 神経疾患 / iPS / 遺伝子 / 治療 / ゲノム / 脳神経疾患 / 発現制御 / トランスレーショナルリサーチ |
Research Abstract |
Epigenetics is a reversible mechanism. Thus, we investigated epigenomic restoration for a representative autistic disorder, Rett syndrome. We initially planned to develop a chemical to restore epigenomic status in a gene-specific manner. However, we realized that genes that require epigenomic restoration were relatively many. Therefore, we searched chemicals with a global effect. As a result, we demonstrated that a drug for epilepsy of Rett syndrome had a global effect with up-regulation of many genes. Furthermore, we identified more effective new chemicals, suggesting that these will be new drug candidates for Rett syndrome when we confirm the effect in our newly established patients-derived iPS cells.
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Report
(4 results)
Research Products
(68 results)
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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, OhoriK, Soutome M, HirasawaT, KazukiY, Adachi N, SuzukiS, ItohM, GotoY, AndohT, KurosawaH, Akamatsu W, Oyama M, Okano H, Oshimura M, Sasaki M, Toyoda A, Kubota T
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Journal Title
Related Report
Peer Reviewed
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[Journal Article] Three novel ZBTB24 mutations identified in Cape Verdean type 2 ICF syndrome patients2013
Author(s)
Nitta H, Unoki M, Ichiyanagi K, Kosho T, Shigemura T, Takahashi H, Velasco G, Franscastel C, Picard C, Kubota T, Sasaki H
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Journal Title
J Hum Genet
Volume: 58
Pages: 455-460
Related Report
Peer Reviewed
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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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[Journal Article] Spread of X-chromosome inactivation into chromosome 15 is associated with Prader-Willi syndrome phenotype in a boy with a t(X;15)(p21.1;q11.2) translocation2012
Author(s)
Sakazume S, OhashiH, SasakiY, Harada N, NakanishiK, SatoH, E m iM, EndohK, SohmaR, Kido Y, NagaiT, KubotaT
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Journal Title
Hum Genet
Volume: 131
Pages: 121-130
Related Report
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[Journal Article] The protocadherins, PCDHB1 a n d PCDH7, are regulated by MeCP2 in neuronal cells and brain tissues : implication for pathogenesis of Rett syndrome2011
Author(s)
Miyake K, Hirasawa T, Soutome M, Itoh M, Goto Y, Endoh K, Takahashi K, Kudo S, Nakawaga T, Yokoi S, Taira T, Inazawa J, Kubota T
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Journal Title
BMC Neurosci
Volume: 12
Pages: 81-81
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[Presentation] Astrocytes are increased in neural cells derived from Rett syndrome iPS cells2013
Author(s)
Ando T, Akamatsu W, Matusmoto, Mikake K, Yamaguchi R, Okada Y, Imaizumi Y, Ohyama M, Kurosawa H, Amagai M, Kubota T, Okano H
Organizer
第35回日本神経科学大会
Place of Presentation
名古屋
Year and Date
2013-07-21
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