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DEC Cell Therapy for DMD Treatment

Duchenne muscular dystrophy (DMD), a lethal disease caused by X-linked mutations of the dystrophin gene, currently lacks effective cures. Researchers led by Maria Siemionow (University of Illinois at Chicago, Chicago, IL, USA) recently developed human dystrophin expressing chimeric (DEC) cells derived from myoblasts of normal and DMD-affected donors as a novel treatment. In their new STEM CELLS Translational Medicine article, Siemionow et al. evaluated their approach after systemic-intraosseous transplantation to an mdx mouse model of DMD and confirmed that DECs improved function and reduced pathology in DMD-affected target organs.