Editors' ChoiceCELL THERAPY

Muscle memory helps with heartache

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Science Translational Medicine  02 Dec 2015:
Vol. 7, Issue 316, pp. 316ec206
DOI: 10.1126/scitranslmed.aad8020

Muscular dystrophies are difficult diseases to treat with gene or cell therapy because patients suffer from loss of both cardiac and skeletal muscles. Stem cell differentiation protocols that make both cardiac and skeletal muscle cells have been established, but thus far no strategy can produce a master muscle cell progenitor that is capable of simultaneously engrafting and regenerating both skeletal and heart muscles. Now, Quattrocelli and co-workers show that it is possible to generate induced pluripotent stem cells (iPSCs) from mouse, dog, or human mesoderm that can produce a muscle progenitor cell that is capable of engrafting and repairing injured skeletal and heart muscle in a dystrophic mouse model.

These investigators used TALEN nucleases to correct a muscular dystrophy mutation in dog mesodermal-derived iPSC muscle progenitor cells ex vivo and then transplanted the corrected cells into skeletal and heart muscle of dystrophic mice. They observed in vivo repair of striatal skeletal and cardiac muscle in the dystrophic animals by the transplanted corrected canine cells. These findings suggest that this strategy could be used for autologous cell therapy whereby mesoderm-derived iPSC muscle progenitor cells from muscular dystrophy patients would first have the mutation corrected ex vivo and then these cells would be transplanted back to the patient for skeletal and cardiac muscle repair. Although this therapeutic strategy still needs further testing and development, it provides a solution to diseases like the muscular dystrophies where more than one cell type is affected.

M. Quattrocelli et al., Mesodermal iPSC–derived progenitor cells functionally regenerate cardiac and skeletal muscle. J. Clin. Invest. 10.1172/JCI82735 (2015). [Full Text]

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