Triptolide: A potential drug for polycystic kidney disease

rmed, as sensed by fluid flow in the tubule. Fluid flow bends the primary cilium, giving a signal to stop cell growth. PKD is caused by a mutation in the PKD1 or PKD2 gene causing the cells to miss the stop growing signal, allowing them to keep growing, and thus generating a cyst.

In collaboration with Stefan Somlo, M.D., Chief of Nephrology at Yale School of Medicine, who discovered the PKD2 gene, triptolide was tested in a mouse model of PKD. Both fewer and smaller kidney cysts formed in mice lacking PKD1 when they were treated with therapeutic amounts of triptolide.

Our research shows that triptolide, an active ingredient of the TCM Lei Gong Teng, markedly decreases cyst formation in a mouse model of this most common genetic lethal kidney disease, Crews said. Our results offer a novel therapeutic approach to this deadly disease and highlight the potential of TCM as pharmaceutical sources.


Contact: Janet Rettig Emanuel
Yale University

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