The anti-leprosy drug clofazimine reduces polyQ toxicity through activation of PPARγ

This is the authors' abstract. We don't add key points for this paper.

BACKGROUND: PolyQ diseases are autosomal dominant neurodegenerative disorders caused by the expansion of CAG repeats. While of slow progression, these diseases are ultimately fatal and lack effective therapies. METHODS: ). Candidate drugs were tested in a wide range of in vitro and in vivo models of polyQ toxicity. FINDINGS: expression. Importantly, clofazimine also limited polyQ toxicity in developing zebrafish and neuron-specific worm models of polyQ disease. INTERPRETATION: Our results support the potential of repurposing the antimicrobial drug clofazimine for the treatment of polyQ diseases. FUNDING: A full list of funding sources can be found in the acknowledgments section.