• Search Research Projects
  • Search Researchers
  • How to Use
  1. Back to previous page

Therapeutic approach for ocular amyloidosis by ocular-targeted multi-functional drug delivery system

Research Project

Project/Area Number 17H06970
Research Category

Grant-in-Aid for Research Activity Start-up

Allocation TypeSingle-year Grants
Research Field Medical pharmacy
Research InstitutionKumamoto University

Principal Investigator

Hayashi Yuya  熊本大学, 医学部附属病院, 特別研究員 (50806429)

Project Period (FY) 2017-08-25 – 2019-03-31
Project Status Completed (Fiscal Year 2018)
Budget Amount *help
¥2,730,000 (Direct Cost: ¥2,100,000、Indirect Cost: ¥630,000)
Fiscal Year 2018: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2017: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Keywords眼アミロイドーシス / ドラッグデリバリーシステム / 核酸医薬 / シクロデキストリン / トランスサイレチン / デンドリマー / DDS / siRNA / アミロイドーシス
Outline of Final Research Achievements

In the present study, we developed ocular-targeted multi-functional siRNA carrier for the therapeutic use of transthyretin (TTR)-related ocular amyloidosis, and evaluated the inhibitory effect of TTR expression and amyloid fibril formation.
Ocular-targeted multi-functional carrier/TTR-siRNA complex elicited TTR silencing effects in vitro and in vivo. Furthermore, ocular-targeted multi-functional siRNA carrier showed potent inhibition of TTR amyloid fibril formation. In conclusion, the present study demonstrated the potentials of ocular-targeted multi-functional siRNA carrier as a novel TTR-related ocular amyloidosis therapy.

Academic Significance and Societal Importance of the Research Achievements

これまでにTTR-FAPにおける全身症状に対しては肝臓移植、内服薬、核酸医薬製剤が開発されたものの、眼アミロイドーシス症状に対する治療法は皆無であり、失明に陥ってしまう多くの患者がいる現状は何としてでも解決しなければならない。本研究で得られた眼特異的多機能型DDSにより眼におけるTTR産生およびアミロイド線維形成を抑制可能という成果は、眼アミロイドーシスの新たな治療法につながる重要な知見であると確信する。

Report

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

    (4 results)

All 2018

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

  • [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 Annual Research Report
    • Peer Reviewed / Open Access
  • [Presentation] TTR-FAP治療を企図した肝臓特異的siRNA・遺伝子キャリアとしてのラクトシル化デンドリマー/シクロデキストリン結合体の構築とその有用性評価2018

    • Author(s)
      林祐也
    • Organizer
      第43回製剤・創剤セミナー
    • Related Report
      2018 Annual Research Report
  • [Presentation] Therapeutic Approach for Transthyretin Amyloidosis by Targeted Delivery of siRNA Ternary Complex with Cyclodextrin/dendrimer Conjugate2018

    • Author(s)
      Yuya Hayashi, Hidetoshi Arima, Yukio Ando, Hideyuki Saito, Hirofumi Jono
    • Organizer
      The XVIth International Symposium on Amyloidosis: ISA2018
    • Related Report
      2017 Annual Research Report
    • Int'l Joint Research

URL: 

Published: 2017-08-25   Modified: 2020-07-01  

Information User Guide FAQ News Terms of Use Attribution of KAKENHI

Powered by NII kakenhi