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.
Program in Developmental and Stem Cell Biology, The Hospital for Sick Children, Toronto Medical Discovery Towers, 101 College Street, Toronto, Ontario, Canada, M5G 1 L7.
Robyn P
RP
Thom
Norman D
ND
Rosenblum
D002648:Child; D053823:Hedgehog Proteins / Q000378:metabolism; D006801:Humans; D006869:Hydronephrosis / Q000151:congenital* / Q000235:genetics / Q000503:physiopathology / Q000628:therapy*; D051741:Kruppel-Like Transcription Factors / Q000378:metabolism; D009419:Nerve Tissue Proteins / Q000378:metabolism; D015398:Signal Transduction; D014551:Urinary Tract / Q000473:pathology; D000074290:Zinc Finger Protein Gli3
C065683:GLI3 protein, human; D053823:Hedgehog Proteins; D051741:Kruppel-Like Transcription Factors; D009419:Nerve Tissue Proteins; D000074290:Zinc Finger Protein Gli3