Development of pathogenesis-based therapy for polyglutamine diseases

点击翻译按钮获取中文摘要

In this study, we demonstrated that the disruption of TGF-β signaling by the pathogenic androgen receptor (AR) is associated with polyglutamine-induced motor neuron damage in SBMA. Our in vivo analyses also showed that Hsp90 inhibitors facilitate proteasomal degradation of the pathogenic AR. These results indicate that pharmacological modification of UPS activity and cellular signals such as TGF-β appear to be promising therapeutic approaches for SBMA and other polyglutamine diseases.