Inheritance of polyalanine expansion mutation of PHOX2B in congenital central hypoventilation syndrome

J Hum Genet. 2012 May;57(5):335-7. doi: 10.1038/jhg.2012.27. Epub 2012 Mar 22.

Abstract

Congenital central hypoventilation syndrome (CCHS; MIM 209880) is caused mostly by dominant alanine expansion (most prevalent is 7-alanine expansion) mutations in PHOX2B. More than 90% of the alanine expansion mutations had been considered to be de novo due to unequal crossover during gametogenesis. However, a recent report stated that 25% of patients inherited the alanine-expanded allele from their parents with somatic mosaicism or constitutive mutation. We studied inheritance in 45 unrelated families, and found that one patient (2%) inherited 5-alanine expansion mutation from a parent with late-onset central hypoventilation syndrome and nine patients (20%) inherited 5- to 7-alanine expansion mutation from apparently asymptomatic parents with somatic mosaicism. Analysis using a sensitive method would be recommended to all parents of CCHS proband due to high incidence of somatic mosaicism. The absence of an alanine-contracted allele (expected counterpart allele in unequal crossover) and the highest prevalence of 6-alanine expansion mutation in somatic mosaicism suggest that the somatic mosaicism is likely caused by a mechanism other than an unequal crossover, such as a replication mechanism.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Family
  • Female
  • Heterozygote
  • Homeodomain Proteins / genetics*
  • Humans
  • Hypoventilation / congenital*
  • Hypoventilation / genetics
  • Male
  • Mosaicism*
  • Mutation / genetics*
  • Peptides / genetics*
  • Sleep Apnea, Central / genetics*

Substances

  • Homeodomain Proteins
  • PHOX2A protein, human
  • Peptides
  • polyalanine

Supplementary concepts

  • Congenital central hypoventilation syndrome