PABPN1 and oculopharyngeal muscular dystrophy: Since the intranuclear aggregates of the mutant PABPN1 protein might be toxic, pharmacological approaches to specifically target these aggregates using cystamine [56], doxycycline [57], trehalose [58], guanabenz [59], and intrabodies [60] successfully ameliorated the disease in several models of OPMD.