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Development of gene therapy using novel double-stranded antisense oligonucleotides

Research Project

Project/Area Number 24659258
Research Category

Grant-in-Aid for Challenging Exploratory Research

Allocation TypeMulti-year Fund
Research Field Applied pharmacology
Research InstitutionTokyo Medical and Dental University

Principal Investigator

NISHINA Kazutaka  東京医科歯科大学, 大学院・医歯学総合研究科, メディカルフェロー (60622529)

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遺伝子診断 / 治療 / アンチセンス核酸 / 核酸医薬 / ドラッグデリバリー
Research Abstract

Here we developed a new class of exceptionally potent antisense oligonucleotides (ASOs) that are hybridized with complementary RNA (cRNA) and conjugated with a ligand. At first, we optimized the chemical modifications of cRNA. Secondly, tocopherol-conjugated ASO (Toc-dsASO) reduced multiple target sequence including mouse apolipoprotein B and human transthyretin. Finally, we showed the liver specific delivery of Toc-dsASO using IVIS imaging system.

Report

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

    (9 results)

All 2013 2012

All Journal Article (3 results) (of which Peer Reviewed: 3 results) Presentation (6 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] Efficient in vivo delivery of antisense oligonucleotide to choroid plexus2013

    • Author(s)
      Wenying Piao, Kazutaka Nishina, KieYoshida-Tanaka, Hiroya Kuwahara, TomokoNishina, Mina Sakata, Hidehiro Mizusawa, Takanori Yokota
    • Journal Title

      Journal of Medical and Dental Sciences

      Volume: vol.60,No.1 Pages: 9-16

    • NAID

      130006943947

    • URL

      http://www.tmd.ac.jp/english/labs/toshozyouhou/lib/6001/index.html

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Efficient in vivo delivery of antisense oligonucleotide to choroid plexus.2013

    • Author(s)
      Wenying Piao, Kazutaka Nishina, Kie Yoshida-Tanaka, Hiroya Kuwahara, Tomoko Nishina, Mina Sakata, Hidehiro Mizusawa, Takanori Yokota
    • Journal Title

      Journal of Medical and Dental Sciences

      Volume: 60 Pages: 9-16

    • NAID

      130006943947

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Presentation] Efficient in vivo delivery of alpha-tocopherol-conjugated siRNA with HDL to the brain2012

    • Author(s)
      Kazutaka Nishina, Yoshitaka Uno, Wenying Piao, Kie Tanaka-Yoshida, Hidehiro Mizusawa, Takanori Yokota
    • Organizer
      8th Annual Meeting of the Oligonucleotide Therapeutics Society
    • Place of Presentation
      The conference center at Harvard Medical School, Boston, USA
    • Year and Date
      2012-10-29
    • Related Report
      2012 Final Research Report
  • [Presentation] アンチセンス核酸の脳脈絡叢へのデリバリー2012

    • Author(s)
      仁科一隆,朴文英,田中規恵,坂田美奈,桑原宏哉,仁科智子,水澤英洋,横田隆徳
    • Organizer
      アンチセンス・遺伝子・デリバリーシンポジウム 2012
    • Place of Presentation
      仙台市民会館
    • Year and Date
      2012-09-24
    • Related Report
      2012 Final Research Report
  • [Presentation] ビタミンE結合siRNAの注腸投与による家族性アミロイドポリニューロパチーの治療法の検討2012

    • Author(s)
      仁科一隆,吉田規恵,桑原宏哉,朴文英,仁科智子,横田隆徳,水澤英洋
    • 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
      2012-05-23
    • Related Report
      2012 Final Research Report
  • [Presentation] Efficient in vivo delivery of alpha-tocopherol-conjugated siRNA with HDL to the brain.2012

    • Author(s)
      Kazutaka Nishina, Yoshitaka Uno, Wenying Piao, Kie Tanaka-Yoshida, Hidehiro Mizusawa, Takanori Yokota.
    • Organizer
      8th Annual Meeting of the Oligonucleotide Therapeutics Society
    • Place of Presentation
      The conference center at Harvard Medical School, Boston
    • Related Report
      2012 Annual Research Report
  • [Presentation] アンチセンス核酸の脳脈絡叢へのデリバリー.2012

    • Author(s)
      仁科 一隆, 朴 文英, 田中 規恵, 坂田 美奈, 桑原 宏哉, 仁科 智子, 水澤 英洋, 横田 隆徳.
    • Organizer
      アンチセンス・遺伝子・デリバリーシンポジウム2012
    • Place of Presentation
      仙台市民会館
    • Related Report
      2012 Annual Research Report
  • [Presentation] ビタミンE結合siRNAの注腸投与による家族性アミロイドポリニューロパチーの治療法の検討.2012

    • Author(s)
      仁科 一隆, 吉田 規恵, 桑原 宏哉, 朴 文英, 仁科 智子, 横田 隆徳, 水澤 英洋.
    • 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
    • Related Report
      2012 Annual Research Report

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

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