Lyonization effects of the t(X;16) translocation on the phenotypic expression in a rare female with Menkes disease

Pediatr Res. 2009 Mar;65(3):347-51. doi: 10.1203/PDR.0b013e3181973b4e.

Abstract

Menkes disease (MD) is a rare and severe X-linked recessive disorder of copper metabolism. The MD gene, ATP7A (ATPase Cu++ transporting alpha polypeptide), encodes an ATP-dependent copper-binding membrane protein. In this report, we describe a girl with typical clinical features of MD, carrying a balanced translocation between the chromosomes X and 16 producing the disruption of one copy of ATP7A gene and the silencing of the other copy because of the chromosome X inactivation. Fluorescence in situ hybridization experiments with bacterial derived artificial chromosome probes revealed that the breakpoints were located within Xq13.3 and 16p11.2. Replication pattern analysis demonstrated that the normal X chromosome was late replicating and consequently inactivated, whereas the der(X)t(X;16), bearing the disrupted ATP7A gene, was active. An innovative approach, based on FMR1 (fragile X mental retardation 1) gene polymorphism, has been used to disclose the paternal origin of the rearrangement providing a new diagnostic tool for determining the parental origin of defects involving the X chromosome and clarifying the mechanism leading to the cytogenetic rearrangement that occurred in our patient.

Publication types

  • Case Reports

MeSH terms

  • Adenosine Triphosphatases / genetics*
  • Cation Transport Proteins / genetics*
  • Cerebral Arteries / pathology
  • Child
  • Chromosomes, Artificial, Bacterial
  • Chromosomes, Human, Pair 16 / genetics*
  • Chromosomes, Human, X / genetics*
  • Copper-Transporting ATPases
  • Female
  • Humans
  • In Situ Hybridization, Fluorescence
  • Karyotyping
  • Menkes Kinky Hair Syndrome / genetics*
  • Menkes Kinky Hair Syndrome / pathology
  • Phenotype*
  • Translocation, Genetic / genetics*
  • X Chromosome Inactivation / genetics*

Substances

  • Cation Transport Proteins
  • Adenosine Triphosphatases
  • ATP7A protein, human
  • Copper-Transporting ATPases