Research Project Outline
Polyglutamine diseases are hereditary disorders including Huntington disease, SBMA and some of hereditary cerebellar ataxias, in which CAG repeat in their responsible genes is expanded. So far, no effective therapy has been developed. In this CREST project, we will develop the comprehensive therapies targeting molecules, which modulate pathological processes including protein degradation, aggregation and gene transcription. We will also employ the methods of chemical genetics, determine the molecular targets and develop new drug and gene therapies.