Research on regenerating muscle fibres using stem cells has been ongoing for decades, particularly in Duchenne muscular dystrophy models, but has unfortunately been unsuccessful. The major problem was the failure of the stem cells to survive and thrive, with endogenous cells, out-competing the donor cells or the cells being rejected by the immune system. In the present study, a highly successful method used in haematopoietic stem cell transplantation was adapted to grow new muscle fibres from stem cells in a mouse model. Professors Peter Gunning and Edna Hardeman of the University of NSW, led the study in which myoblasts expressing a chemotherapy-resistant gene were transplanted into mice, with the host muscle ablated by chemotherapy. When the endogenous cells were destroyed, the muscle stem cells began to proliferate and muscle fibre formation as well as significant regeneration of satellite cells were observed. This procedure overcomes one of the major barriers to stem cell therapy, i.e. allowing the cells to survive for more than an hour after introduction into damaged tissue. Even though the procedure is still in its pre-clinical stage, the authors aim to launch human trials treating specific forms of muscular dystrophy, such as oculopharyngeal dystrophy, within the next three to five years.

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