Abstract
An important corollary to the recent advances in our understanding of the primary cause of Duchenne muscular dystrophy1–6, is the validation of genuine genetic homologues as animal models of the disease7,8 in which potential therapies can be tested. The persistent skeletal muscle necrosis that characterizes human Duchenne muscular dystrophy9 is also seen in the mdx mouse10–13 and is, in both, a consequence of a deficiency of dystrophin6,7, probably within the muscle fibres themselves14–16. As injected muscle precursor cells of one genotype can fuse with host muscle fibres of a different genotype and express the donor genes17,18, we decided to test grafts of normal muscle precursor cells to see if they could induce synthesis of dystrophin in innately dystrophin-deficient mdx muscle fibres. We show that injected normal muscle precursor cells can fuse with pre-existing or regenerating mdx muscle fibres to render many of these fibres dystrophin-positive and so to partially or wholly rescue them from their biochemical defect.
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Partridge, T., Morgan, J., Coulton, G. et al. Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts. Nature 337, 176–179 (1989). https://doi.org/10.1038/337176a0
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DOI: https://doi.org/10.1038/337176a0
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