Triptolide reduces cystogenesis in a model of ADPKD

SJ Leuenroth, N Bencivenga, P Igarashi… - Journal of the …, 2008 - journals.lww.com
Mutations in PKD1 result in autosomal dominant polycystic kidney disease, which is
characterized by increased proliferation of tubule cells leading to cyst initiation and
subsequent expansion. Given the cell proliferation associated with cyst growth, an attractive
therapeutic strategy has been to target the hyperproliferative nature of the disease. We
previously demonstrated that the small molecule triptolide induces cellular calcium release
through a polycystin-2–dependent pathway, arrests Pkd1−/− cell growth, and reduces cystic …