The DUX4 gene associated with Facioscapulohumeral muscular dystrophy (FSHD) encodes a transcription factor protein that is toxic when expressed in numerous organisms. We hypothesized that one avenue for developing an FSHD treatment could involve inhibiting DUX4 protein activity. However, little is known about the mechanisms that may confer toxic properties to the DUX4 protein. We hypothesized that characterizing the biochemical properties of the DUX4 protein would provide fundamental information required to ultimately design FSHD therapies aimed at inhibiting DUX4 toxicity at the protein level.

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