Cell based therapy for duchenne muscular dystrophy

Authors

  • Andrea Farini,

    1. Stem Cell Laboratory, Department of Neurological Science, Centro Dino Ferrari, University of Milan, Fondazione IRCCS Policlinico Mangiagalli Regina Elena, Italy
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  • Paola Razini,

    1. Stem Cell Laboratory, Department of Neurological Science, Centro Dino Ferrari, University of Milan, Fondazione IRCCS Policlinico Mangiagalli Regina Elena, Italy
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  • Silvia Erratico,

    1. Stem Cell Laboratory, Department of Neurological Science, Centro Dino Ferrari, University of Milan, Fondazione IRCCS Policlinico Mangiagalli Regina Elena, Italy
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  • Yvan Torrente,

    Corresponding author
    1. Stem Cell Laboratory, Department of Neurological Science, Centro Dino Ferrari, University of Milan, Fondazione IRCCS Policlinico Mangiagalli Regina Elena, Italy
    • Department of Neurological Science, University of Milan, Padiglione Ponti, Fondazione IRCCS Policlinico Mangiagalli Regina Elena, via Francesco Sforza 35, 20122 Milan, Italy.
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  • Mirella Meregalli

    1. Stem Cell Laboratory, Department of Neurological Science, Centro Dino Ferrari, University of Milan, Fondazione IRCCS Policlinico Mangiagalli Regina Elena, Italy
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Abstract

Mutations in the dystrophin gene cause an X-linked genetic disorder: Duchenne muscular dystrophy (DMD). Stem cell therapy is an attractive method to treat DMD because a small number of cells are required to obtain a therapeutic effect. Here, we discussed about multiple types of myogenic stem cells and their possible use to treat DMD. The identification of a stem cell population providing efficient muscle regeneration is critical for the progression of cell therapy for DMD. We speculated that the most promising possibility for the treatment of DMD is a combination of different approaches, such as gene and stem cell therapy. J. Cell. Physiol. 221: 526–534, 2009. © 2009 Wiley-Liss, Inc.

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