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My research goal is to identify compounds and molecules that interfere with activation of the FSHD-causing gene, DUX4. By preventing DUX4 from being upregulated during muscle formation, we may be able to prevent further muscle wasting in patients with the disease. During my first round of funding from the Friends of FSH research, I devised strategies to measure DUX4 and DUX4-induced muscle death in a cell culture model. I have used the model to determine that activation of a developmentally regulated signaling cascade can prevent DUX4 from being activated in cultured muscle. My most recent proposal capitalizes on these findings and will allow me to further dissect the mechanisms usually keeping DUX4 at bay. Cultured muscle has its limitations; however, any good discovery pipeline requires this kind of assay to move forward into larger animal models and clinical trials. As we continue to develop and validate our cell culture model, we are confident we will be identify novel strategies to combat the disease.
— Gregory J Block