Mammalian Models of Duchenne Muscular Dystrophy: Pathological Characteristics and Therapeutic Applications

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Abstract

Duchenne muscular dystrophy (DMD) is a devastating X-linked muscle disorder characterized by muscle wasting which is caused by mutations in the DMD gene. The DMD gene encodes the sarcolemmal protein dystrophin, and loss of dystrophin causes muscle degeneration and necrosis. Thus far, therapies for this disorder are unavailable. However, various therapeutic trials based on gene therapy, exon skipping, cell therapy, read through therapy, or pharmaceutical agents have been conducted extensively. In the development of therapy as well as elucidation of pathogenesis in DMD, appropriate animal models are needed. Various animal models of DMD have been identified, and mammalian (murine, canine, and feline) models are indispensable for the examination of the mechanisms of pathogenesis and the development of therapies. Here, we review the pathological features of DMD and therapeutic applications, especially of exon skipping using antisense oligonucleotides and gene therapies using viral vectors in murine and canine models of DMD.

Journal

  • JOURNAL OF BIOMEDICINE AND BIOTECHNOLOGY

    JOURNAL OF BIOMEDICINE AND BIOTECHNOLOGY (2011), 184393, 2011

    HINDAWI PUBLISHING CORPORATION

Codes

  • NII Article ID (NAID)
    120005440221
  • NII NACSIS-CAT ID (NCID)
    AA11698349
  • Text Lang
    ENG
  • Article Type
    journal article
  • Data Source
    IR 
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