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Journal of Biomedicine and Biotechnology
Volume 2011 (2011), Article ID 184393, 8 pages
http://dx.doi.org/10.1155/2011/184393
Review Article

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

1Department of Medicine (Neurology and Rheumatology), School of Medicine Shinshu University, 3-1-1 Ahahi, Matsumoto 390-8621, Japan
2Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-higashi, Kodaira, Tokyo 187-8502, Japan

Received 14 October 2010; Accepted 19 December 2010

Academic Editor: Andrea Vecchione

Copyright © 2011 Akinori Nakamura and Shin'ichi Takeda. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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.