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

Research output: Contribution to journalArticle

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.

Original languageEnglish
Article numbere61
JournalCell Death and Disease
Volume1
Issue number8
DOIs
Publication statusPublished - Aug 2010

Keywords

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

ASJC Scopus subject areas

  • Cell Biology
  • Immunology
  • Cancer Research
  • Cellular and Molecular Neuroscience
  • Medicine(all)

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