Genetic analysis of BIRC4/XIAP as a putative modifier gene of Wilson disease

J Inherit Metab Dis. 2010 Dec:33 Suppl 3:S233-40. doi: 10.1007/s10545-010-9123-5. Epub 2010 Jun 2.

Abstract

Wilson disease (WD) is an autosomal-recessive copper overload disorder caused by mutations in the copper-transporting adenosine triphosphatase (ATPase) ATP7B. It presents with a highly variable clinical phenotype ranging from asymptomatic to fulminant hepatic failure or progressive neurological involvement. No clear genotype-phenotype correlation has been established. Thus, variants in modifier genes could have an impact on WD manifestation and severity. Recently, the antiapoptotic protein baculoviral IAP repeat-containing protein 4 BIRC4/XIAP has been suggested as a regulator of copper-induced cell death. With the aim of investigating a putative role of BIRC4/XIAP as modifier gene in individuals with copper overload, we analyzed a WD patient cohort (n = 98) for sequence variants at the BIRC4/XIAP locus. When compared with clinical data, the previously described coding single nucleotide polymorphisms (SNPs) at the BRIC4/XIAP locus (rs28382721, rs28382722, rs28382723, rs5956583, rs28382740, rs12838858, rs28382741) did not correlate with age of onset or clinical presentation in our collective. However, three previously unreported variants in the BIRC4/XIAP gene were identified (c.1-26 T > G; c.1408A > T; p.T470S; c.1019A > G; p.N340S). The two patients with variants leading to amino acid exchanges in the BIRC4/XIAP protein showed a remarkably early disease onset at the age of 5 years. Furthermore, one of these patients was only heterozygous for disease-causing mutations in the ATP7B gene. In summary, these data emphasize the need to further elucidate a role of BIRC4/XIAP variants as putative pathogenetic factors in copper overload disorders.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenosine Triphosphatases / genetics*
  • Adolescent
  • Adult
  • Age of Onset
  • Cation Transport Proteins / genetics*
  • Chi-Square Distribution
  • Child
  • Child, Preschool
  • Copper-Transporting ATPases
  • DNA Mutational Analysis
  • Female
  • Gene Frequency
  • Genes, Modifier*
  • Genetic Predisposition to Disease
  • Hepatolenticular Degeneration / diagnosis
  • Hepatolenticular Degeneration / genetics*
  • Heterozygote
  • Humans
  • Male
  • Middle Aged
  • Mutation*
  • Phenotype
  • Polymorphism, Single Nucleotide*
  • Risk Factors
  • X-Linked Inhibitor of Apoptosis Protein / genetics*
  • Young Adult

Substances

  • Cation Transport Proteins
  • X-Linked Inhibitor of Apoptosis Protein
  • XIAP protein, human
  • Adenosine Triphosphatases
  • ATP7B protein, human
  • Copper-Transporting ATPases