We found that administration of GW4869 (5 g/g) (n = 5) delayed cyst growth (Supplementary Fig. 13a, b) as seen by the decrease of cyst index, KW/BW ratio, and BUN level (Supplementary Fig. 13c–e), and cystic epithelial cell proliferation (Supplementary Fig. 13f) in kidneys from Pkd1flox/flox: Pkhd1-Cre mice compared to controls. This evidence concerns the gene PKHD1 and cyst.