Therapeutic strategy for the polyglutamine diseases by selective degradation of expanded polyglutamine proteins using polyglutamine-binding pepetides

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Toward developing a therapy for the polyglutamine (polyQ) neurodegenerative diseases by specific degradation of expanded polyQ proteins, 1) we showed that p62 plays an important role in the autophagic clearance of polyQ protein, especially its oligomers, resulting in protection against polyQ-induced neurodegeneration in Drosophila. 2) We further designed the p62-QBP1 chimeric protein, which consists of p62, an adaptor protein for selective autophagy and the expanded polyQ-specific binding peptide QBP1, and demonstrated its therapeutic potential for the polyQ diseases by selective degradation of expanded polyQ proteins.