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2013 Fiscal Year Final Research Report

Development of novel gene therapy targeting age-related macular degeneration

Research Project

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Project/Area Number 23659813
Research Category

Grant-in-Aid for Challenging Exploratory Research

Allocation TypeMulti-year Fund
Research Field Ophthalmology
Research InstitutionNagasaki University

Principal Investigator

SASAKI Hitoshi  長崎大学, 大学病院, 教授 (00170689)

Project Period (FY) 2011 – 2013
Keywords加齢黄斑変性症 / 遺伝子治療
Research Abstract

In this study, we developed the safe and efficient novel ocular gene delivery system to the age-related macular degeneration. We successfully prepared the stable nanoparticles (pDNA-PEI complex: PEI complex, pDNA-PEI-gamma-PGA complex: PGA complex, pDNA-PEI-CS complex: CS complex) by optimization of the mix ratio. The PGA complex and CS complex showed high gene expression comparable to PEI complex. While PEI complex showed severe agglutination of vitreous body and cytotoxicity, PGA complex and CS complex showed no agglutination of vitreous body and no cytotoxicity. We also developed siRNA-PEI-gamma-PGA (siPGA complexes). The siPGA complexes showed high silencing effect comparable to commercial vector (lipofectamine) without cytotoxicity.

  • Research Products

    (16 results)

All 2013 2012 2011

All Journal Article (6 results) (of which Peer Reviewed: 6 results) Presentation (10 results)

  • [Journal Article] Secure splenic delivery of plasmid DNA and its application to DNA vaccine2013

    • Author(s)
      Kurosaki T, Kodama Y, Muro T, Higuchi N, Nakamura T, Kitahara T, Miyakoda M, Yui K, Sasaki H
    • Journal Title

      Biol Pharm Bull

      Volume: 36(11) Pages: 1800-6

    • Peer Reviewed
  • [Journal Article] Ternary complex of plasmid DNA with protamine and γ-polyglutamic acid for biocompatible gene delivery system2013

    • Author(s)
      Kanda K, Kodama Y, Kurosaki T, Imamura M, Nakagawa H, Muro T, Higuchi N, Nakamura T, Kitahara T, Honda M, Sasaki H
    • Journal Title

      Biol Pharm Bull

      Volume: 36(11) Pages: 1794-9

    • Peer Reviewed
  • [Journal Article] Ocular gene delivery systems using ternary complexes of plasmid DNA, polyethylenimine, and anionic polymers2013

    • Author(s)
      Kurosaki T, Uematsu M, Shimoda K, Suzuma K, Nakai M, Nakamura T, Kitahara T, Kitaoka T, Sasaki H
    • Journal Title

      Biol Pharm Bull

      Volume: 36(1) Pages: 96-101

    • Peer Reviewed
  • [Journal Article] Self-assemble gene delivery system for molecular targeting using nucleic acid aptamer2012

    • Author(s)
      Kurosaki T, Higuchi N, Kawakami S, Higuchi Y, Nakamura T, Kitahara T, Hashida M, Sasaki H
    • Journal Title

      Gene

      Volume: 491(2) Pages: 205-9

    • Peer Reviewed
  • [Journal Article] Secure and effective gene vector of polyamidoamine dendrimer pharmaceutically modified with anionic polymer2011

    • Author(s)
      Kurosaki T, Yamashita Y, Aki K, Harasawa H, Nakagawa H, Kodama Y, Higuchi N, Nakamura T, Kitahara T, Sasaki H
    • Journal Title

      J Pharm Sci

      Volume: 100(11) Pages: 4855-63

    • Peer Reviewed
  • [Journal Article] Nanoparticles electrostatically coated with folic acid for effective gene therapy2011

    • Author(s)
      Kurosaki T, Morishita T, Kodama Y, Sato K, Nakagawa H, Higuchi N, Nakamura T, Hamamoto T, Sasaki H, Kitahara T
    • Journal Title

      Mol Pharm

      Volume: 8(3) Pages: 913-9

    • Peer Reviewed
  • [Presentation] 樹状細胞標的型ナノデバイスを用いたマラリアDNA ナノワクチンの開発2013

    • Author(s)
      西垣 和香、兒玉 幸修、北原 隆志、佐々木 均
    • Organizer
      第30回日本薬学会九州支部大会
    • Place of Presentation
      佐世保
    • Year and Date
      2013-12-08
  • [Presentation] Dendrigraft poly-L-lysine を用いた生体内分解型遺伝子ベクターの開発2013

    • Author(s)
      塩川 裕美、兒玉 幸修、北原 隆志、佐々木 均
    • Organizer
      日本薬剤学会第28年会
    • Place of Presentation
      名古屋
    • Year and Date
      2013-05-25
  • [Presentation] ポリヌクレオチド被膜型複合体による脾臓指向性新規遺伝子ベクターの開発2013

    • Author(s)
      矢次 結衣子、兒玉 幸修、大久保 智佳子、北原 隆志、佐々木 均
    • Organizer
      日本薬剤学会第28年会
    • Place of Presentation
      名古屋
    • Year and Date
      2013-05-25
  • [Presentation] Fetuin 被膜型複合体による新規遺伝子ベクターの構築2013

    • Author(s)
      兒玉 幸修、北原 隆志、江頭 かの子、中嶋 幹郎、樋口 則英、中村 忠博、佐々木 均
    • Organizer
      日本薬学会第133年会
    • Place of Presentation
      横浜
    • Year and Date
      2013-03-28
  • [Presentation] 静電的自己組織化を基盤としたアニオン性siRNA ベクターの開発2012

    • Author(s)
      塩川 裕美、兒玉 幸修、原内 智慧、北原 隆志、佐々木 均
    • Organizer
      第29回日本薬学会九州支部大会
    • Place of Presentation
      熊本
    • Year and Date
      2012-12-08
  • [Presentation] 安全かつ効果的な眼疾患用新規遺伝子ベクターの開発2012

    • Author(s)
      佐々木 均、兒玉 幸修、黑﨑 友亮、樋口 則英、北原 隆志、中村 忠博
    • Organizer
      第32回日本眼薬理学会
    • Place of Presentation
      滋賀
    • Year and Date
      2012-09-15
  • [Presentation] ホスファチジルセリンアナログを用いた脾臓指向性遺伝子導入ベクターの開発2012

    • Author(s)
      大久保 智佳子、仲宗根 ちひろ、黒崎 友亮、兒玉 幸修、北原 隆志、佐々木 均
    • Organizer
      日本薬剤学会第27年会
    • Place of Presentation
      神戸
    • Year and Date
      2012-05-24
  • [Presentation] 肝細胞選択的なグリチルリチン含有遺伝子ベクターの開発2012

    • Author(s)
      原内 智慧、川鍋 早紀、黒崎 友亮、兒玉 幸修、北原 隆志、佐々木 均
    • Organizer
      日本薬剤学会第27年会
    • Place of Presentation
      神戸
    • Year and Date
      2012-05-24
  • [Presentation] 制癌剤と遺伝子同時送達を目的としたメトトレキサート被膜型遺伝子ベクターの開発2012

    • Author(s)
      兒玉 幸修、黒崎 友亮、江頭 かの子、中嶋 幹郎、中川 博雄、樋口 則英、中村 忠博、北原 隆志、佐々木 均
    • Organizer
      日本薬学会第132年会
    • Place of Presentation
      札幌
    • Year and Date
      2012-03-31
  • [Presentation] Polyplex of pDNA with poly-L-lysine, poly-L-histidine, and γ-polyglutamic acid for biocompatible gene delivery system2011

    • Author(s)
      Yukinobu Kodama, Tomoaki Kurosaki, Kanoko Egashira, Tadahiro Nakamura, Takashi Kitahara, Hitoshi Sasaki
    • Organizer
      日本薬物動態学会第26回年会
    • Place of Presentation
      広島
    • Year and Date
      2011-11-17

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Published: 2015-06-25  

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