Therapeutic strategy for polyglutamine-mediated motor neuron disease via molecular chaperone-ubiquitin proteasome system

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We administrated the peony extract to male spinal and bulbar muscular atrophy (SBMA) transgenic mouse model (androgen receptor (AR)-97Q or AR-24Q) and examined various neurological and behavioral parameters. Administration of the peony extract markedly ameliorated motor impairments in AR-97Q mice without detectable toxicity, and reduced amounts of monomeric and nuclear accumulated mutant AR. Mutant AR was preferentially degraded in the presence of the compound in both SBMA cell and mouse models when compared with wild-type AR. It also significantly induced Hsp70 and Hsp40. These observations suggest that the compound is a promising therapeutic candidate for polyglutamine (polyQ)-mediated neurodegenerative diseases including SBMA.