A mouse with an N-Ethyl-N-nitrosourea (ENU) Induced Trp589Arg Galnt3 mutation represents a model for hyperphosphataemic familial tumoural calcinosis

PLoS One. 2012;7(8):e43205. doi: 10.1371/journal.pone.0043205. Epub 2012 Aug 13.

Abstract

Mutations of UDP-N-acetyl-alpha-D-galactosamine polypeptide N-acetyl galactosaminyl transferase 3 (GALNT3) result in familial tumoural calcinosis (FTC) and the hyperostosis-hyperphosphataemia syndrome (HHS), which are autosomal recessive disorders characterised by soft-tissue calcification and hyperphosphataemia. To facilitate in vivo studies of these heritable disorders of phosphate homeostasis, we embarked on establishing a mouse model by assessing progeny of mice treated with the chemical mutagen N-ethyl-N-nitrosourea (ENU), and identified a mutant mouse, TCAL, with autosomal recessive inheritance of ectopic calcification, which involved multiple tissues, and hyperphosphataemia; the phenotype was designated TCAL and the locus, Tcal. TCAL males were infertile with loss of Sertoli cells and spermatozoa, and increased testicular apoptosis. Genetic mapping localized Tcal to chromosome 2 (62.64-71.11 Mb) which contained the Galnt3. DNA sequence analysis identified a Galnt3 missense mutation (Trp589Arg) in TCAL mice. Transient transfection of wild-type and mutant Galnt3-enhanced green fluorescent protein (EGFP) constructs in COS-7 cells revealed endoplasmic reticulum retention of the Trp589Arg mutant and Western blot analysis of kidney homogenates demonstrated defective glycosylation of Galnt3 in Tcal/Tcal mice. Tcal/Tcal mice had normal plasma calcium and parathyroid hormone concentrations; decreased alkaline phosphatase activity and intact Fgf23 concentrations; and elevation of circulating 1,25-dihydroxyvitamin D. Quantitative reverse transcriptase-PCR (qRT-PCR) revealed that Tcal/Tcal mice had increased expression of Galnt3 and Fgf23 in bone, but that renal expression of Klotho, 25-hydroxyvitamin D-1α-hydroxylase (Cyp27b1), and the sodium-phosphate co-transporters type-IIa and -IIc was similar to that in wild-type mice. Thus, TCAL mice have the phenotypic features of FTC and HHS, and provide a model for these disorders of phosphate metabolism.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Apoptosis / genetics
  • Blotting, Western
  • Bone and Bones / metabolism
  • COS Cells
  • Calcinosis / genetics*
  • Calcinosis / pathology*
  • Chlorocebus aethiops
  • Disease Models, Animal*
  • Ethylnitrosourea / toxicity
  • Fibroblast Growth Factor-23
  • Fibroblast Growth Factors / metabolism
  • Genes, Recessive / genetics
  • Green Fluorescent Proteins / genetics
  • Green Fluorescent Proteins / metabolism
  • Hyperostosis, Cortical, Congenital / genetics*
  • Hyperostosis, Cortical, Congenital / pathology*
  • Hyperphosphatemia / genetics*
  • Hyperphosphatemia / pathology*
  • Male
  • Mice
  • Mutation, Missense / drug effects
  • Mutation, Missense / genetics*
  • N-Acetylgalactosaminyltransferases / genetics*
  • N-Acetylgalactosaminyltransferases / metabolism
  • Polypeptide N-acetylgalactosaminyltransferase
  • Reverse Transcriptase Polymerase Chain Reaction
  • Sertoli Cells / pathology
  • Spermatozoa / pathology
  • Testis / cytology

Substances

  • Fgf23 protein, mouse
  • enhanced green fluorescent protein
  • Green Fluorescent Proteins
  • Fibroblast Growth Factors
  • Fibroblast Growth Factor-23
  • N-Acetylgalactosaminyltransferases
  • Ethylnitrosourea

Supplementary concepts

  • Tumoral Calcinosis, Hyperphosphatemic, Familial