A translational approach to congenital non-obstructive hydronephrosis.

Congenital hydronephrosis, defined as congenital dilatation of one or more components of the renal collecting system, is detected in 1-2% of all pregnancies. The majority of antenatally detected hydronephrosis is non-obstructive and either resolves or stabilizes. Management of persistently severe cases may include surgical intervention, the only available treatment. Recent data demonstrate that hedgehog signaling plays a critical role in regulating the structure and function of the collecting system in a manner dependent on the formation of GLI3 repressor. Here, we review the pathobiology and clinical management of non-obstructive hydronephrosis and describe how inhibitors of GLI3 repressor formation may serve as novel therapies for this disorder.