Progressive myopathy in an inducible mouse model of oculopharyngeal muscular dystrophy

► We generated mifepristone-inducible transgenic mouse model of OPMD ► Inducible expression of mutant PABPN1 resulted in skeletal and cardiac myopathy ► De-induction of mutant PABPN1 attenuated the myopathy and reduced PABPN1 aggregates ► Silencing the expression of mutant PABPN1 may be an option for therapy in OPMD