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Drug screen identifies leflunomide for treatment of inflammatory bowel diseases caused by TTC7A deficiency

Gastroenterology Nov 24, 2019

Jardine S, Anderson S, Babcock S, et al. - Considering that the intestinal disease is severe, untreatable, and recurs in patients with tetratricopeptide repeat domain 7A gene (TTC7A) deficiency despite resection or allogeneic hematopoietic stem cell transplant, researchers sought to screen drugs for those that prevent apoptosis of in cells with TTC7A deficiency and test their impacts in an animal model of the disease. According to findings, leflunomide was identified as an agent in a drug screen that decreases apoptosis and levels of caspase 3 and activates AKT signaling and in TTC7A-knockout cells. Leflunomide restores gut motility in zebrafish with disruption of ttc7a, decreases intestinal tract narrowing, and increases survival of the intestinal cell. This drug may be repurposed to treat the deficiency of TTC7A.
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