The Development of an Ultrasound-mediated Nucleic Acid Delivery System for Treating Muscular Dystrophies

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  • Negishi Yoichi
    Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences
  • Hamano Nobuhito
    Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences
  • Shiono Hitomi
    Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences
  • Akiyama Saki
    Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences
  • Endo-Takahashi Yoko
    Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences
  • Suzuki Ryo
    Laboratory of Drug and Gene Delivery, Faculty of Pharma Sciences, Teikyo University
  • Maruyama Kazuo
    Laboratory of Drug and Gene Delivery, Faculty of Pharma Sciences, Teikyo University
  • Aramaki Yukihiko
    Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences

Bibliographic Information

Other Title
  • 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
  • キンジストロフィー チリョウ ニ ムケタ チョウオンパ カクサン デリバリー システム ノ カイハツ

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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, 2012-12-01

    The Pharmaceutical Society of Japan

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