VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects

摘要: Absence of dystrophin makes skeletal muscle more susceptible to injury, resulting in breaches the plasma membrane and chronic inflammation Duchenne muscular dystrophy (DMD). Current management by glucocorticoids has unclear molecular benefits harsh side effects. It is uncertain whether therapies that avoid hormonal stunting growth development, and/or immunosuppression, would be or less beneficial. Here, we discover an oral drug with mechanisms provide efficacy through anti-inflammatory signaling membrane-stabilizing pathways, independent immunosuppressive We find VBP15 protects promotes efficient repair cells upon laser opposition prednisolone. Potent inhibition NF-κB mediated protein interactions glucocorticoid receptor, however shows significantly reduced receptor transcriptional activity. The translation these into DMD model mice improves strength, live-imaging pathology both preventive post-onset intervention regimens. These data demonstrate successful improvement hormonal, growth, effects, indicating merits clinical investigation for benefit other inflammatory diseases.