Aberrant activation of the mammalian target of rapamycin (mTOR) pathway occurs in
polycystic kidney disease (PKD). mTOR inhibitors, such as rapamycin, are highly effective in
several rodent models of PKD, but these models result from mutations in genes other than
Pkd1 and Pkd2, which are the primary genes responsible for human autosomal dominant
PKD. To address this limitation, we tested the efficacy of rapamycin in a mouse model that
results from conditional inactivation of Pkd1. Mosaic deletion of Pkd1 resulted in PKD and …