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Development of novel triple-stranded antisense oligonucleotides

Research Project

Project/Area Number 24659044
Research Category

Grant-in-Aid for Challenging Exploratory Research

Allocation TypeMulti-year Fund
Research Field Drug development chemistry
Research InstitutionTokyo Medical and Dental University

Principal Investigator

YOKOTA Takanori  東京医科歯科大学, 大学院・医歯学総合研究科, 教授 (90231688)

Project Period (FY) 2012
Project Status Completed (Fiscal Year 2012)
Budget Amount *help
¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000)
Fiscal Year 2012: ¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000)
Keywords医薬分子設計 / アンチセンス / 核酸医薬 / デリバリー / 後根神経節細胞 / ELISA / RT-PCR / PNA / ホスホロチオエート結合修飾
Research Abstract

Here we developed a new class of exceptionally potent antisense oligonucleotides (ASOs) that are hybridized with complementary RNA (cRNA) strand and peptide-conjugated PNA strand. At first, we optimized the chemical modifications and length of the strands of the triple-stranded ASO (tsASO). Secondly, peptide-conjugated tsASO reduced mouse TRPV1 gene in dorsal root ganglia (DRG). Finally, we showed the delivery of DRG-delivered peptide-conjugated tsASO using confocal imaging and quantitative RT-PCR by systemic administration.

Report

(2 results)
  • 2012 Annual Research Report   Final Research Report ( PDF )
  • Research Products

    (3 results)

All 2013 2012

All Journal Article (2 results) (of which Peer Reviewed: 2 results) Presentation (1 results)

  • [Journal Article] Short interfering RNA and the central nervous system: development of nonviral delivery systems2013

    • Author(s)
      Kazutaka Nishina, Hidehiro Mizusawa, Takanori Yokota
    • Journal Title

      Expert Opin Drug Deliv

      Volume: vol.10,No.3 Issue: 3 Pages: 289-292

    • DOI

      10.1517/17425247.2013.748746

    • Related Report
      2012 Annual Research Report 2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Intrathecal shRNA-AAV9 inhibits target protein expression in the spinal cord and dorsal root ganglia of adult mice.2012

    • Author(s)
      Hirai T, Enomoto M, Machida A, Yamamoto M, Kuwahara H, Tajiri M, Hirai Y, Sotome S, Mizusawa H, Shinomiya K, Okawa A, Yokota T.
    • Journal Title

      Hum Gene Ther Methods

      Volume: 23 Issue: 2 Pages: 119-27

    • DOI

      10.1089/hgtb.2012.035

    • Related Report
      2012 Annual Research Report 2012 Final Research Report
    • Peer Reviewed
  • [Presentation] 神経疾患に対する核酸医薬による遺伝子治療2012

    • Author(s)
      横田隆徳
    • Organizer
      第53回日本神経学会学術大会
    • Place of Presentation
      東京国際フォーラム
    • Year and Date
      2012-05-25
    • Related Report
      2012 Annual Research Report 2012 Final Research Report

URL: 

Published: 2013-05-31   Modified: 2019-07-29  

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