• Mashup Score: 8

    One protein, Piezo1, is key to marshalling muscle stem cells’ unique shapes and response to injuries, but it is in low supply in those with Duchenne muscular dystrophy, according to a team at the Perelman School of Medicine at the University of Pennsylvania. However, when they re-activated Piezo1, it allowed muscle stem cells in mice to return to their normal, distinctly-shaped states so that…

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    • Piezo1 possible key to supporting #muscle regeneration in Duchenne #MuscularDystrophy @pennmedicine @ScienceAdvances https://t.co/7alkPcXBWV

  • Mashup Score: 1

    Author summary Oculopharyngeal muscular dystrophy (OPMD) is a genetic disease characterized by progressive weakness of specific muscles, leading to swallowing difficulties (dysphagia), eyelid drooping (ptosis) and walking difficulties at later stages. No drug treatments are currently available. OPMD is due to mutations in a nuclear protein called poly(A) binding protein nuclear 1 (PABPN1) that is…

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    • There are no current drug treatments for oculopharyngeal #MuscularDystrophy Ribot et al show in a #Drosophila model of OPMD, oral treatment with a proteasome inhibitor reduces muscle defects Similar inhibitors are already approved for cancer treatment https://t.co/avrbEcc110