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Potential use of multifunctional polyamidoamine Dendrimer for curative treatments of intractable amyloidoses

Research Project

Project/Area Number 17K19498
Research Category

Grant-in-Aid for Challenging Research (Exploratory)

Allocation TypeMulti-year Fund
Research Field Pharmaceutical Sciences and related fields
Research InstitutionKumamoto University

Principal Investigator

JONO Hirofumi  熊本大学, 医学部附属病院, 准教授 (40515483)

Co-Investigator(Kenkyū-buntansha) 安東 由喜雄  熊本大学, 大学院生命科学研究部(医), 教授 (20253742)
有馬 英俊  熊本大学, 大学院生命科学研究部(薬), 教授 (50260964)
Project Period (FY) 2017-06-30 – 2019-03-31
Project Status Completed (Fiscal Year 2018)
Budget Amount *help
¥6,500,000 (Direct Cost: ¥5,000,000、Indirect Cost: ¥1,500,000)
Fiscal Year 2018: ¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000)
Fiscal Year 2017: ¥2,600,000 (Direct Cost: ¥2,000,000、Indirect Cost: ¥600,000)
Keywords高機能性遺伝子キャリア / RNAi医薬 / アミロイドーシス / 家族性アミロイドポリニューロパチー / 樹状高分子デンドリマー
Outline of Final Research Achievements

Amyloidoses, specified rare and intractable diseases, are thought to result from conformational change and aggregation of precursor proteins. Accumulation of amyloid fibrils, which are produced via self-assembly of a naturally folded protein into an insoluble cross-β-sheet structure, is considered to be the hallmark of amyloid-related diseases. It is well-documented that amyloidoses progress through 3 critical pathological processes, such as, 1. Overproduction or mutation of amyloid precursor proteins, 2. Amyloid fibril formation caused by comformational change, 3. Tissue deposition of amyloid fibrils. In this research, we utilize characteristic features of polyamidoamine starburst dendrimer as a novel multi-functional gene transfer carrier and develop novel multi-target drugs for intractable amyloidoses.

Academic Significance and Societal Importance of the Research Achievements

特定疾患に指定され臨床上大きな問題となっている難治性アミロイドーシスには、アミロイド原因タンパク質の違いによって、TTR関連アミロイドーシスやアルツハイマー病など様々な疾患が含まれており、その治療薬開発は喫緊の社会的課題である。本研究により実証された、樹状高分子化合物デンドリマーの新規治療薬としての有効性・適応拡大性は、多様なアミロイドーシスへの根治療法開発に大きく貢献することが期待される。

Report

(3 results)
  • 2018 Annual Research Report   Final Research Report ( PDF )
  • 2017 Research-status Report
  • Research Products

    (10 results)

All 2019 2018 Other

All Int'l Joint Research (1 results) Journal Article (3 results) (of which Peer Reviewed: 3 results,  Open Access: 2 results) Presentation (6 results) (of which Int'l Joint Research: 3 results,  Invited: 3 results)

  • [Int'l Joint Research] Alnylam Pharmaceuticals, Inc.(米国)

    • Related Report
      2017 Research-status Report
  • [Journal Article] Therapeutic Potential of Polyamidoamine Dendrimer for Amyloidogenic Transthyretin Amyloidosis2019

    • Author(s)
      Inoue M, Ueda M, Higashi T, Anno T, Fujisawa K, Motoyama K, Mizuguchi M, Ando Y, Jono H, Arima H.
    • Journal Title

      ACS Chemical Neuroscience

      Volume: 印刷中 Issue: 5 Pages: 2584-2590

    • DOI

      10.1021/acschemneuro.9b00059

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Recent Advances in Oligonucleotide-Based Therapy for Transthyretin Amyloidosis: Clinical Impact and Future Prospects2018

    • Author(s)
      Hayashi Yuya、Jono Hirofumi
    • Journal Title

      Biological and Pharmaceutical Bulletin

      Volume: 41 Issue: 12 Pages: 1737-1744

    • DOI

      10.1248/bpb.b18-00625

    • NAID

      130007539542

    • ISSN
      0918-6158, 1347-5215
    • Year and Date
      2018-12-01
    • Related Report
      2018 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] In vitro and in vivo siRNA delivery to hepatocyte utilizing ternary complexation of lactosylated dendrimer/cyclodextrin conjugates, siRNA and low-molecular-weight sacran2018

    • Author(s)
      Hayashi Yuya、Higashi Taishi、Motoyama Keiichi、Jono Hirofumi、Ando Yukio、Arima Hidetoshi
    • Journal Title

      International Journal of Biological Macromolecules

      Volume: 107 Pages: 1113-1121

    • DOI

      10.1016/j.ijbiomac.2017.09.085

    • Related Report
      2017 Research-status Report
    • Peer Reviewed / Open Access
  • [Presentation] 臨床化学的観点を活用した遺伝性 ATTR アミロイドーシスの新規治療戦略の確立2019

    • Author(s)
      城野博史、齋藤秀之、安東由喜雄
    • Organizer
      日本薬学会第139年会
    • Related Report
      2018 Annual Research Report
    • Invited
  • [Presentation] 質量分析を活用したアミロイドーシスの病型診断2018

    • Author(s)
      城野博史、田崎雅義、大林光念、安東由喜雄
    • Organizer
      第43回日本医用マススペクトル学会年会
    • Related Report
      2018 Annual Research Report
    • Invited
  • [Presentation] 難治性アミロイドーシスに対する薬物治療の現状と今後の展望2018

    • Author(s)
      城野博史、安東由喜雄、齋藤秀之
    • Organizer
      第28回日本医療薬学会年会
    • Related Report
      2018 Annual Research Report
    • Invited
  • [Presentation] Effects of Cyclodextrin/Dendrimer Conjugate/shRNA Complex as a Novel Multi-target Drug on Amyloidoses2018

    • Author(s)
      Jono H, Inoue M, Hayashi Y, Ouchi K, Muta K, Higashi T, Motoyama K, Arima H, Ando Y
    • Organizer
      International Symposia on Amyloidosis 2018
    • Related Report
      2017 Research-status Report
    • Int'l Joint Research
  • [Presentation] Therapeutic Approach for Transthyretin Amyloidosis by Targeted Delivery of siRNA Ternary Complex with Cyclodextrin/dendrimer Conjugate2018

    • Author(s)
      Hayashi Y, Arima H, Ando Y, Saito H, Jono H
    • Organizer
      International Symposia on Amyloidosis 2018
    • Related Report
      2017 Research-status Report
    • Int'l Joint Research
  • [Presentation] Evaluation of Cyclodextrin/Dendrimer Conjugate/shRNA Complex for Treatment of Transthyretin Amyloidosis2018

    • Author(s)
      Inoue M, Jono H, Higashi T, Motoyama K, Ando Y, Arima H
    • Organizer
      International Symposia on Amyloidosis 2018
    • Related Report
      2017 Research-status Report
    • Int'l Joint Research

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Published: 2017-07-21   Modified: 2022-02-21  

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