Mutations in PITX2 may contribute to cases of omphalocele and VATER-like syndromes

Am J Med Genet A. 2004 Oct 15;130A(3):277-83. doi: 10.1002/ajmg.a.30329.

Abstract

Omphalocele is a congenital anomaly with substantial morbidity. Rieger syndrome, an autosomal dominant disorder, is characterized by craniofacial abnormalities and abdominal wall defects. PITX2 mutations are etiologic in >40% of cases of Rieger syndrome. We demonstrate that the birth prevalence of omphalocele is significantly higher in Rieger syndrome than in the general population, with omphaloceles found in 0.03% in the Iowa newborn population and 4.3% of patients with Rieger syndrome. Our objective was to screen coding and conserved non-coding regions of PITX2 for mutations in 209 patients with omphalocele. We identified remarkable evolutionarily conserved regions by comparing the 3'UTR of Pitx2 in 13 vertebrate and 3 invertebrate species. No mutations changing the amino acid sequence were found within the omphalocele population. In one case of omphalocele with VATER-like additional anomalies, a three nucleotide deletion was found in the 3'UTR. This deletion was not seen in 1,186 controls. Also in the 3'UTR, we identified a single nucleotide polymorphism at a highly conserved residue. Our findings suggest additional studies of PITX2 conserved regions will be valuable. We also screened the omphalocele cases for mutations in exon 5 of the gene FLNA. Mutations in FLNA have been shown to cause a broad range of congenital malformations, including otopalatodigital syndrome type 2 in which a missense mutation occurring in exon 5 of FLNA results in omphalocele as part of the phenotype. We did not find any mutations in exon 5 of FLNA in 179 omphalocele cases studied.

Publication types

  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Abnormalities, Multiple / genetics*
  • Abnormalities, Multiple / pathology
  • Animals
  • Base Sequence
  • Conserved Sequence / genetics
  • DNA Mutational Analysis
  • Evolution, Molecular
  • Hernia, Umbilical / genetics*
  • Hernia, Umbilical / pathology
  • Homeobox Protein PITX2
  • Homeodomain Proteins / genetics*
  • Humans
  • Infant
  • Molecular Sequence Data
  • Mutation*
  • Sequence Alignment
  • Sequence Homology, Nucleic Acid
  • Syndrome
  • Transcription Factors / genetics*

Substances

  • Homeodomain Proteins
  • Transcription Factors