Two novel nonsense mutations in GALNT3 gene are responsible for familial tumoral calcinosis

J Hum Genet. 2007;52(5):464-468. doi: 10.1007/s10038-007-0126-5. Epub 2007 Mar 10.

Abstract

Ectopic periarticular calcifications associated with elevated levels of serum phosphate represent the principal clinical features of hyperphosphatemic familial tumoral calcinosis (HFTC), a rare autosomal recessive metabolic disorder. The disease can be caused by recessive mutations in at least two different genes: GalNAc transferase 3 (GALNT3), encoding a glycosyltransferase that initiates mucin-type O-glycosylation, and fibroblast growth factor 23 (FGF23), which encodes a regulator of phosphate circulating levels. In the current study, we performed mutation analyses of the GALNT3 gene in a subject with HFTC and in his relatives. Sequence analyses revealed that the proband was a compound heterozygote for two novel nonsense mutations in exon 4 (Y322X) and in exon 7 (Q481X). Cosegregation of the mutations with the disease within the family was confirmed by polymerase chain reaction restriction fragment length polymorphism (PCR-RFLP) analysis. This is the first report describing the simultaneous presence of two different stop codons in the coding sequence of the GALNT3 gene.

MeSH terms

  • Calcinosis / genetics*
  • Codon, Nonsense*
  • DNA Mutational Analysis
  • Exons
  • Female
  • Fibroblast Growth Factor-23
  • Humans
  • Hypophosphatemia, Familial / genetics*
  • Italy
  • Male
  • N-Acetylgalactosaminyltransferases / genetics*
  • Neoplasm Proteins / genetics*
  • Pedigree
  • Polypeptide N-acetylgalactosaminyltransferase

Substances

  • Codon, Nonsense
  • FGF23 protein, human
  • Neoplasm Proteins
  • Fibroblast Growth Factor-23
  • N-Acetylgalactosaminyltransferases