Inhibition of Hedgehog signaling suppresses proliferation and microcyst formation of human Autosomal Dominant Polycystic Kidney Disease cells

Sci Rep. 2018 Mar 21;8(1):4985. doi: 10.1038/s41598-018-23341-2.

Abstract

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is caused by mutation of PKD1 or PKD2, which encode polycystin 1 and 2, respectively. The polycystins localize to primary cilia and the functional loss of the polycystin complex leads to the formation and progressive growth of fluid-filled cysts in the kidney. The pathogenesis of ADPKD is complex and molecular mechanisms connecting ciliary dysfunction to renal cystogenesis are unclear. Primary cilia mediate Hedgehog signaling, which modulates cell proliferation and differentiation in a tissue-dependent manner. Previously, we showed that Hedgehog signaling was increased in cystic kidneys of several PKD mouse models and that Hedgehog inhibition prevented cyst formation in embryonic PKD mouse kidneys treated with cAMP. Here, we show that in human ADPKD tissue, Hedgehog target and activator, Glioma 1, was elevated and localized to cyst-lining epithelial cells and to interstitial cells, suggesting increased autocrine and paracrine Hedgehog signaling in ADPKD, respectively. Further, Hedgehog inhibitors reduced basal and cAMP-induced proliferation of ADPKD cells and cyst formation in vitro. These data suggest that Hedgehog signaling is increased in human ADPKD and that suppression of Hedgehog signaling can counter cellular processes that promote cyst growth in vitro.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Aged
  • Animals
  • Benzamides / pharmacology
  • Benzimidazoles / pharmacology
  • Cell Differentiation / drug effects
  • Cell Proliferation / drug effects
  • Cells, Cultured
  • Cilia / metabolism
  • Cilia / pathology*
  • Cyclohexylamines / pharmacology
  • Epithelial Cells
  • Hedgehog Proteins / antagonists & inhibitors*
  • Hedgehog Proteins / metabolism
  • Humans
  • Kidney / cytology
  • Kidney / pathology*
  • Mice
  • Middle Aged
  • Polycystic Kidney, Autosomal Dominant / genetics
  • Polycystic Kidney, Autosomal Dominant / pathology*
  • Primary Cell Culture
  • Pyridines / pharmacology
  • Pyrimidines / pharmacology
  • Signal Transduction / drug effects
  • TRPP Cation Channels / genetics
  • TRPP Cation Channels / metabolism
  • Thiophenes / pharmacology
  • Up-Regulation
  • Zinc Finger Protein GLI1 / metabolism

Substances

  • Benzamides
  • Benzimidazoles
  • Cyclohexylamines
  • GANT 61
  • GLI1 protein, human
  • Hedgehog Proteins
  • Pyridines
  • Pyrimidines
  • SAG compound
  • SANT-2 compound
  • TRPP Cation Channels
  • Thiophenes
  • Zinc Finger Protein GLI1
  • polycystic kidney disease 1 protein
  • polycystic kidney disease 2 protein