Mechanism of Action and Therapeutic Route for a Muscular Dystrophy Caused by a Genetic Defect in Lipid Metabolism

Treatment of Chkb−/− myocytes with peroxisome proliferator-activated receptor agonists enabled fatty acids to be used for β-oxidation and prevented triacyglyerol accumulation, while simultaneously increasing expression of the compensatory choline kinase alpha isoform, preventing muscle cell injury.