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

J. Díaz-Manera, T. Touvier, A. Dellavalle, R. Tonlorenzi, F. S. Tedesco, G. Messina, M. Meregalli, C. Navarro, L. Perani, C. Bonfanti, I. Illa, Y. Torrente, G. Cossu

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41 Citations (Scopus)

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. © 2010 Macmillan Publishers Limited.
Original languageEnglish
Article numbere61
JournalCell Death and Disease
Volume1
Issue number8
DOIs
Publication statusPublished - 1 Aug 2010

Keywords

  • A/J mice
  • Dysferlin
  • Mesoangioblasts
  • Stem cells
  • Therapy

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