Neurotoxic effects of androgens in spinal and bulbar muscular atrophy

► Spinobulbar muscular atrophy (SBMA) is caused by CAG expansion in androgen receptor. ► SBMA is triggered by the binding of mutant androgen receptor to testosterone. ► Protein context/structure dictates disease features. ► Alteration of the native function of the disease protein is critical for pathogenesis. ► Towards development of therapy for SBMA.