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Partial dysferlin reconstitution by adult murine mesoangioblasts is sufficient for full functional recovery in a murine model of dysferlinopathy

2010; Springer Nature; Volume: 1; Issue: 8 Linguagem: Inglês

10.1038/cddis.2010.35

2041-4889

Jordi Díaz‐Manera, Thierry Touvier, Arianna Dellavalle, Rossana Tonlorenzi, Francesco Saverio Tedesco, Graziella Messina, Mirella Meregalli, Claire Navarro, Laura Perani, Chiara Bonfanti, Isabel Illa, Yvan Torrente, Giulio Cossu,

Genetics, Aging, and Longevity in Model Organisms

Dysferlin deficiency leads to a peculiar form of muscular dystrophy due to a defect in sarcolemma repair and currently lacks a therapy. We developed a cell therapy protocol with wild-type adult murine mesoangioblasts. These cells differentiate with high efficiency into skeletal muscle in vitro but differ from satellite cells because they do not express Pax7. After intramuscular or intra-arterial administration to SCID/BlAJ mice, a novel model of dysferlinopathy, wild-type mesoangioblasts efficiently colonized dystrophic muscles and partially restored dysferlin expression. Nevertheless, functional assays performed on isolated single fibers from transplanted muscles showed a normal repairing ability of the membrane after laser-induced lesions; this result, which reflects gene correction of an enzymatic rather than a structural deficit, suggests that this myopathy may be easier to treat with cell or gene therapy than other forms of muscular dystrophies.