Targeting muscle stem cell intrinsic defects to treat Duchenne muscular dystrophy

Abstract excerpt

gene that encodes for the protein dystrophin. Dystrophin has critical roles in myofiber stability and integrity by connecting the actin cytoskeleton to the extracellular matrix. Absence of dystrophin leads to myofiber fragility and contributes to skeletal muscle degeneration in DMD patients, however, accumulating evidence also indicate that muscle stem cells (also known as satellite cells) are defective in...

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