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Duchenne Muscular Dystrophy (DMD) is an incurable progressive myopathy, caused by the absence of the dystrophin protein. It is characterized by permanent myofiber necrosis, leading to continuous activation of muscle stem cells (MuSCs), that eventually cannot support muscle repair leading to replacement of the muscle by fibro/fatty tissue. We have observed that epigenetic changes in human MuSCs from DMD patients contribute extensively to reduced muscle regeneration. Taking advantage of the complementary skills and expertise of the French and Canadian labs, the proposed project aims to prevent loss of muscle regeneration in DMD
by restoring the healthy epigenetic state in the diseased MuSCs. Importantly, the proteins that control the epigenetic state are potential targets for intervention using small molecule drugs. Thus, the knowledge gained from these studies will be important to the development of new drug therapies to treat muscle degeneration in DMD.
Jeffrey Dilworth
Université Claude Bernard Lyon 1
Life Sciences
Education
University of Ottawa
Globalink Research Award
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