Mutations in the gene TTC7A cause intestinal and epithelial defects and a severe early onset intestinal disease. Using a cell-based drug screen and validation in a zebrafish model of disease Aleixo Muise and colleagues discovered that the FDA-approved drug Leflunomide may reverse cell and organ defects seen in TTC7A disease. In collaboration with Jay Thiagarajah and colleagues Leflunomide was found to also reverse changes in patient-derived cells, suggesting that the drug may be repurposed for the treatment of TTC7A disease.
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Jardine et al Gastro 2020