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Establishment of tailor-made RNAi knockdown technique against triplet repeat disease-causing alleles

Research Project

Project/Area Number 21890312
Research Category

Grant-in-Aid for Research Activity Start-up

Allocation TypeSingle-year Grants
Research Field General medical chemistry
Research InstitutionNational Center of Neurology and Psychiatry

Principal Investigator

TAKAHASHI Masaki  独立行政法人国立精神・神経医療研究センター, 神経研究所・神経薬理研究部, 流動研究員 (00549529)

Project Period (FY) 2009 – 2010
Project Status Completed (Fiscal Year 2010)
Budget Amount *help
¥2,639,000 (Direct Cost: ¥2,030,000、Indirect Cost: ¥609,000)
Fiscal Year 2010: ¥1,248,000 (Direct Cost: ¥960,000、Indirect Cost: ¥288,000)
Fiscal Year 2009: ¥1,391,000 (Direct Cost: ¥1,070,000、Indirect Cost: ¥321,000)
Keywords発現制御 / RNAi治療 / SNP / 脳神経疾患 / 対立遺伝子 / トリプレットリピート病
Research Abstract

We present determination of the coding SNP (cSNP) haplotypes of triplet repeat disease-causing alleles by a novel pull-down method, and demonstrate disease-allele specific RNAi targeting such cSNP sites in the mutant Huntingtin alleles, each of which possesses a different cSNP haplotype. Thus, our procedures allow for allele-specific RNAi against disease-causing alleles by using appropriate siRNAs as the disease-linked cSNP haplotype demands. Therefore, our current progress paves the way to the achievement of a tailor-made RNAi treatment for triplet repeat diseases and other diseases using cSNPs as RNAi targets.

Report

(3 results)
  • 2010 Annual Research Report   Final Research Report ( PDF )
  • 2009 Annual Research Report
  • Research Products

    (12 results)

All 2011 2010 2009 Other

All Journal Article (2 results) (of which Peer Reviewed: 2 results) Presentation (4 results) Book (2 results) Remarks (1 results) Patent(Industrial Property Rights) (3 results)

  • [Journal Article] Tailor-made RNAi knockdown against triplet repeat disease-causing alleles2010

    • Author(s)
      Takahashi M, Watanabe S, Murata M, Furuya H, Kanazawa I, Wada K, Hohjoh H
    • Journal Title

      Proc Natl Acad Sci USA. 107(50)

      Pages: 21731-21736

    • Related Report
      2010 Final Research Report
    • Peer Reviewed
  • [Journal Article] Tailor-made RNAi knockdown against triplet repeat disease-causing alleles2010

    • Author(s)
      Takahashi M, Watanabe S, Murata M, Furuya H, Kanazawa I, Wada K, Hohjoh H
    • Journal Title

      Proc Natl Acad Sci USA.

      Volume: 107 Pages: 21731-21736

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Presentation] 新規プルダウン法を用いたリピート病原因対立遺伝子の優先的回収法とSNPを指標とした病因対立遺伝子の特異的RNAiノックダウン2010

    • Author(s)
      高橋理貴、渡邉荘子、村田美穂、古谷博和、金澤一郎、和田圭司、北條浩彦
    • Organizer
      分子生物学会
    • Place of Presentation
      兵庫県神戸国際会議場
    • Year and Date
      2010-12-10
    • Related Report
      2010 Annual Research Report
  • [Presentation] Identification of triplet repeat disease-causing alleles by a novel pull-down method and disease-causing allele-specific silencing by RNAi2010

    • Author(s)
      Takahashi M, Watanabe S, Murata M, Furuya H, Kanazawa I, Wada K, Hohjoh H
    • Organizer
      ASHG(American Society of Human Genetics)
    • Place of Presentation
      アメリカワシントンDCワシントンコンベンションセンター
    • Year and Date
      2010-11-03
    • Related Report
      2010 Annual Research Report
  • [Presentation] 新規プルダウン法を用いたリピート病原因対立遺伝子の優先的回収法とSNPを指標とした病因対立遺伝子の特異的RNAiノックダウン2010

    • Author(s)
      高橋理貴、渡邉荘子、村田美穂、古谷博和、金澤一郎、和田圭司、北條浩彦
    • Organizer
      分子生物学会
    • Place of Presentation
      神戸国際展示場
    • Related Report
      2010 Final Research Report
  • [Presentation] Identification of triplet repeat disease-causing alleles by a novel pull-down method and disease-causing allele-specific silencing by RNAi2010

    • Author(s)
      高橋理貴, 渡邉荘子, 村田美穂, 古谷博和, 金澤一郎, 和田圭司, 北條浩彦
    • Organizer
      ASHG
    • Place of Presentation
      ワシントンコンベンションセンター
    • Related Report
      2010 Final Research Report
  • [Book] トリプレット・リピート病原因遺伝子に対するテーラーメイドRNAiノックダウン.(29)2011

    • Author(s)
      高橋理貴、北條浩彦
    • Publisher
      「mTOR シグナル」実験医学
    • Related Report
      2010 Final Research Report
  • [Book] 実験医学(「mTORシグナル」 : トリプレットリピート病原因遺伝子に対するテーラーメイドRNAiノックダウン)2011

    • Author(s)
      高橋理貴、北條浩彦
    • Total Pages
      4
    • Publisher
      羊土社
    • Related Report
      2010 Annual Research Report
  • [Remarks] 本研究成果はプレスリリースされ、産経新聞や他数誌で掲載された。

    • Related Report
      2010 Final Research Report
  • [Patent(Industrial Property Rights)] 優性変異遺伝子発現抑制剤2010

    • Inventor(s)
      北條浩彦、高橋理貴
    • Industrial Property Rights Holder
      独立行政法人国立精神・神経医療研究センター
    • Industrial Property Number
      2010-222847
    • Filing Date
      2010-09-30
    • Related Report
      2010 Annual Research Report 2010 Final Research Report
  • [Patent(Industrial Property Rights)] 優性アレル発現抑制剤2010

    • Inventor(s)
      北條浩彦、高橋理貴
    • Industrial Property Rights Holder
      独立行政法人国立精神・神経医療研究センター
    • Industrial Property Number
      2010-139925
    • Filing Date
      2010-06-18
    • Related Report
      2010 Annual Research Report 2010 Final Research Report
  • [Patent(Industrial Property Rights)] 長鎖繰返し配列を含有する遺伝子又は遺伝子産物の選択的又は優先的回収方法2009

    • Inventor(s)
      北條浩彦、高橋理貴
    • Industrial Property Rights Holder
      独立行政法人国立精神・神経医療研究センター
    • Industrial Property Number
      2009-283653
    • Filing Date
      2009-12-15
    • Related Report
      2010 Final Research Report 2009 Annual Research Report

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Published: 2009-04-01   Modified: 2016-04-21  

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