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 language | English |
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Article number | e61 |
Journal | Cell Death and Disease |
Volume | 1 |
Issue number | 8 |
DOIs | |
Publication status | Published - 1 Aug 2010 |
Keywords
- A/J mice
- Dysferlin
- Mesoangioblasts
- Stem cells
- Therapy