Drug development for polyglutamine diseases by a combined phenotype analysis system

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We developed strategically a combined model system for analyzing phenotypes and pathomechanisms and available for developing novel therapeutics of polyglutamine diseases. The system consisted of cell, fly and mouse models was very powerful when combined with omics analyses. We could elucidate a new pathomechanism of polyglutamine diseases, DNA repair impairment and could identify key molecules such as HMGB, Ku70, Hsp70, Omi and Maxer. Furthermore, we identified candidate drugs for polyglutamine diseases by using the combined analysis system.