PKHD1 and cyst: To extend the translational significance of our in vivo findings to an early-stage ADPKD mouse model, we also administrated GW4869 to Pkd1flox/flox:Pkhd1-Cre mice, an aggressive ADPKD mouse model, in which the Cre is driven by the Pkhd1 promoter that results in cyst formation by postnatal day 9 or 1044.