筋ジストロフィー治療に向けた超音波核酸デリバリーシステムの開発  [in Japanese] The Development of an Ultrasound-mediated Nucleic Acid Delivery System for Treating Muscular Dystrophies  [in Japanese]

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Author(s)

    • 根岸 洋一 Negishi Yoichi
    • 東京薬科大学薬学部薬物送達学教室 Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences
    • 濱野 展人 Hamano Nobuhito
    • 東京薬科大学薬学部薬物送達学教室 Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences
    • 塩野 瞳 [他] Shiono Hitomi
    • 東京薬科大学薬学部薬物送達学教室 Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences
    • 秋山 早希 Akiyama Saki
    • 東京薬科大学薬学部薬物送達学教室 Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences
    • 高橋(遠藤) 葉子 Endo-Takahashi Yoko
    • 東京薬科大学薬学部薬物送達学教室 Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences
    • 鈴木 亮 Suzuki Ryo
    • 帝京大学薬学部薬物送達学研究室 Laboratory of Drug and Gene Delivery, Faculty of Pharma Sciences, Teikyo University
    • 丸山 一雄 Maruyama Kazuo
    • 帝京大学薬学部薬物送達学研究室 Laboratory of Drug and Gene Delivery, Faculty of Pharma Sciences, Teikyo University
    • 新槇 幸彦 Aramaki Yukihiko
    • 東京薬科大学薬学部薬物送達学教室 Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences

Abstract

  Muscular dystrophies are a group of heterogeneous diseases that are characterized by progressive muscle weakness, wasting and degeneration. These muscular deficiencies are often caused by the loss of the protein dystrophin, a crucial element of the dystrophin-glycoprotein complex of muscle fibers. Duchenne muscular dystrophy (DMD) is a fatal, X-linked muscular disease that occurs in 1 out of every 3500 males. Therefore, feasible strategies for replacing or repairing the defective gene are required; however, to date, no effective therapeutic strategies for muscular dystrophies have been established. In this review, we first introduce gene therapies mediated by adeno-associated viruses (AAVs) including a functional dystrophin cDNA or antisense oligonucleotide (AO)-induced exon-skipping therapies, which are designed to exclude the mutated or additional exon(s) in the defective gene and thereby correct the translational reading frame. Recently, we developed “Bubble liposomes” (BLs), which are polyethylene glycol (PEG)-modified liposomes entrapping echo-contrast gas that is known as ultrasound (US) imaging gas. BL application combined with US exposure can function as a novel gene delivery tool, and we demonstrate that the US-mediated eruption of BLs is a feasible and efficient technique to deliver plasmid DNA or AOs for the treatment of muscular dystrophies.<br>

Journal

  • YAKUGAKU ZASSHI

    YAKUGAKU ZASSHI 132(12), 1383-1388, 2012

    The Pharmaceutical Society of Japan

Codes

  • NII Article ID (NAID)
    130002489535
  • NII NACSIS-CAT ID (NCID)
    AN00284903
  • Text Lang
    JPN
  • ISSN
    0031-6903
  • NDL Article ID
    024129263
  • NDL Call No.
    Z19-411
  • Data Source
    NDL  J-STAGE 
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