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Development of Comprehensive Therapies for Polyglutamine Diseases

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.

Research Director
Lab Head, RIKEN
Research Started
Research Area
Creation of a Novel Technology towards Diagnosis and Treatment based on Understanding of Molecular Pathogenesis of Psychiatric and Neurological Disorders
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