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They chose to mainly target DUX4 exon 3, which is specifically associated with the pathogenic DUX4 transcript (see DUX4 gene expression)
Neurochem Res 37:26132626 Hohnholt MC, Andersen VH, Andersen JV, Christensen SK, Karaca M, Maechler P, Waagepetersen HS (2018) Glutamate dehydrogenase is essential to sustain neuronal oxidative energy metabolism during stimulation
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Insulin receptor substrate and p55 orthologous adaptor proteins function in the Caenorhabditis elegans daf-2/insulin-like signaling pathway
