Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard
Nantes
France

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The Role of Stem Cells in Muscular Dystrophies

Author(s): Mirella Meregalli, Andrea Farini, Federica Colleoni, Letizia Cassinelli, Yvan Torrente.

Abstract:

Muscular dystrophies are heterogeneous neuromuscular disorders of inherited origin, including Duchenne muscular dystrophy (DMD). Cell-based therapies were used to promote muscle regeneration with the hope that the host cells repopulated the muscle and improved muscle function and pathology. Stem cells were preferable for therapeutic applications, due to their capacity of self-renewal and differentiative potential. In the last years, encouraging results were obtained with adult stem cells to treat muscular dystrophies. Adult stem cells were found into various tissues of the body and they were able to maintain, generate, and replace terminally differentiated cells within their own specific tissue because of cell turnover or tissue injury. Moreover, it became clear that these cells could participate into regeneration of more than just their resident organ. Here, we described multiple types of muscle and non muscle-derived myogenic stem cells, their characterization and their possible use to treat muscular dystrophies. We also underlined that most promising possibility for the management and therapy of DMD is a combination of different approaches, such as gene and stem cell therapy.

Keywords: Adult stem cells, cell therapy, DMD, gene therapy, iPS cell, muscular dystrophies, CD133+, hSM-MSCs, exon, GRMD

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Article Details

VOLUME: 12
ISSUE: 3
Year: 2012
Page: [192 - 205]
Pages: 14
DOI: 10.2174/156652312800840559
Price: $58