Current advances in cell therapy strategies for muscular dystrophies

Abstract

Introduction: Muscular dystrophies are a heterogeneous group of genetic diseases characterized by muscle weakness, wasting and degeneration. Cell therapy consists of delivering myogenic precursor cells to damaged tissue for the complementation of missing proteins and/or the regeneration of new muscle fibres.

Areas covered: We focus on human candidate cells described so far (myoblasts, mesoangioblasts, pericytes, myoendothelial cells, CD133⁺ cells, aldehyde-dehydrogenase-positive cells, mesenchymal stem cells, embryonic stem cells, induced pluripotent stem cells), gene-based strategies developed to modify cells prior to injection, animal models (dystrophic and/or immunodeficient) used for pre-clinical studies, and clinical trials that have been performed using cell therapy strategies. The approaches are reviewed in terms of feasibility, hurdles, potential solutions and/or research areas from where the solution may come and potential application in terms of types of dystrophies and targets.

Expert opinion: Cell therapy for muscular dystrophies should be put in the context of which dystrophy or muscle group is targeted, what tools are available at hand, but even more importantly what can cell therapy bring as compared with and/or in combination with other therapeutic strategies. The solution will probably be the right dosage of these combinations adapted to each dystrophy, or even to each type of mutation within a dystrophy.

Keywords::

• animal model
• cell therapy
• gene therapy
• muscular dystrophy
• regenerative medicine
• skeletal muscle
• stem cell
• transplantation

Acknowledgements

The authors wish to thank Anne Bigot, the MSG study group, and all members of the laboratory for fruitful discussions. They also wish to thank Thanasis Laliotis for the artwork production, and Lidia Dolle for technical assistance.

Notes

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