Regulation and activity of the DUX4 transcription factor

Investigators: Stephen Tapscott MD PhD, Amy E Campbell PhD

Category: Research - Basic

This award provides support for professional dedicated lab personnel with exceptional expertise in FSHD research to both perform research and to train new investigators, graduate students, postdoctoral fellows, and collaborators seeking to pursue FSHD research projects. Specific projects include clinical studies to identify biomarkers for disease activity and progression that might be used in future clinical trials; generation of mouse models that might improve preclinical studies of FSHD therapeutics; determining how drugs can block the activity of DUX4 as the basis for developing new therapeutics for FSHD; and determining how DUX4-induced changes in protein stability contributes to cell toxicity and disease.

Related Material

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See Publication Quantitative proteomics reveals key roles for post-transcriptional gene regulation in the molecular pathology of facioscapulohumeral muscular dystrophy

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See To make itself heard, DUX4 steals the mic and turns the noise down

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