A Phex mutation in a murine model of X-linked hypophosphatemia alters phosphate responsiveness of bone cells

J Bone Miner Res. 2012 Feb;27(2):453-60. doi: 10.1002/jbmr.544.

Abstract

Mutations in the PHEX gene cause X-linked hypophosphatemia (XLH). Hypophosphatemia in XLH results from increased circulating levels of a phosphaturic hormone, fibroblast growth factor 23 (FGF23), which inhibits renal phosphate reabsorption and 1,25-dihydroxyvitamin D (calcitriol) synthesis. The current standard therapy for XLH--high-dose phosphate and calcitriol--further increases FGF23 concentrations, suggesting that patients with XLH may have an altered response to extracellular phosphate. To test for the presence of abnormal phosphate responsiveness, we compared serum biochemistries and femoral Fgf23 mRNA expression between wild-type mice, murine models of XLH (Phex(K496X)) and hyperphosphatemic tumoral calcinosis (Galnt3(-/-)), and Galnt3/Phex double-mutant mice. Phex mutant mice had not only increased Fgf23 expression but also reduced proteolytic cleavage of intact Fgf23 protein, resulting in markedly elevated intact Fgf23 levels and consequent hypophosphatemia. In contrast, despite markedly increased Fgf23 expression, Galnt3 knockout mice had significantly high proteolytic cleavage of Fgf23 protein, leading to low intact Fgf23 concentrations and hyperphosphatemia. Galnt3/Phex double-mutant mice had an intermediate biochemical phenotype between wild-type and Phex mutant mice, including slightly elevated intact Fgf23 concentrations with milder hypophosphatemia. Despite the hypophosphatemia, double-mutant mice attempted to reduce serum phosphate back to the level of Phex mutant mice by upregulating Fgf23 expression as much as 24-fold higher than Phex mutant mice. These data suggest that Phex mutations alter the responsiveness of bone cells to extracellular phosphate concentrations and may create a lower set point for "normal" phosphate levels.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Disease Models, Animal
  • Familial Hypophosphatemic Rickets / blood
  • Familial Hypophosphatemic Rickets / enzymology*
  • Familial Hypophosphatemic Rickets / genetics*
  • Female
  • Femur / drug effects
  • Femur / metabolism
  • Femur / pathology
  • Fibroblast Growth Factor-23
  • Fibroblast Growth Factors / blood
  • Fibroblast Growth Factors / genetics
  • Gene Expression Regulation / drug effects
  • Genetic Diseases, X-Linked*
  • Male
  • Mice
  • Mice, Knockout
  • Mutation / genetics*
  • N-Acetylgalactosaminyltransferases / deficiency
  • N-Acetylgalactosaminyltransferases / metabolism
  • Osteocytes / drug effects*
  • Osteocytes / metabolism*
  • Osteocytes / pathology
  • PHEX Phosphate Regulating Neutral Endopeptidase / genetics*
  • PHEX Phosphate Regulating Neutral Endopeptidase / metabolism
  • Phenotype
  • Phosphates / pharmacology*
  • Polypeptide N-acetylgalactosaminyltransferase
  • Protein Processing, Post-Translational / drug effects
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism

Substances

  • Fgf23 protein, mouse
  • Phosphates
  • RNA, Messenger
  • Fibroblast Growth Factors
  • Fibroblast Growth Factor-23
  • N-Acetylgalactosaminyltransferases
  • PHEX Phosphate Regulating Neutral Endopeptidase
  • Phex protein, mouse