DMD – ANIMAL MODELS & PRECLINICAL TREATMENT

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Cardiosphere-derived cells (CDCs) are cardiac progenitor/stromal cells with immunomodulatory, anti-fibrotic, and pro-regenerative properties. These therapeutic actions antagonize crucial pathways central to the pathology of Duchenne muscular dystrophy (DMD). We have previously demonstrated that CDCs are therapeutic in Duchenne muscular dystrophy (DMD) patients and in mdx mice. Our earlier preclinical work centered on the infusion of a single dose of CDCs to aged mdx mice. Due to the progressive nature of DMD, we questioned whether repeated CDC infusion to mdx mice, beginning in youth, could prevent disease progression into adulthood.

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