Partial dysferlin reconstitution by adult murine mesoangioblasts is sufficient for full functional recovery in a murine model of dysferlinopathy
Articolo
Data di Pubblicazione:
2010
Citazione:
Partial dysferlin reconstitution by adult murine mesoangioblasts is sufficient for full functional recovery in a murine model of dysferlinopathy / J. Dıaz-Manera, T. Touvier, A. Dellavalle, R. Tonlorenzi, F.S. Tedesco, G. Messina, M. A. Meregalli, C. Navarro, L. Perani, C. Bonfanti, I. Illa, Y. Torrente, G. Cossu. - In: CELL DEATH & DISEASE. - ISSN 2041-4889. - 1:8(2010 Aug 05), pp. e61.e61-e61.e61. [10.1038/cddis.2010.35]
Abstract:
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.
Tipologia IRIS:
01 - Articolo su periodico
Keywords:
A/J mice; Dysferlin; Mesoangioblasts; Stem cells; Therapy
Elenco autori:
J. Dıaz Manera, T. Touvier, A. Dellavalle, R. Tonlorenzi, F.S. Tedesco, G. Messina, M.A. Meregalli, C. Navarro, L. Perani, C. Bonfanti, I. Illa, Y. Torrente, G. Cossu
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