Cerebrotendinous xanthomatosis in a Hong Kong Chinese kinship with a novel splicing site mutation IVS6-1G>T in the sterol 27-hydroxylase gene

Mol Genet Metab. 2004 Feb;81(2):144-6. doi: 10.1016/j.ymgme.2003.11.005.

Abstract

We reported a Hong Kong Chinese proband with Cerebrotendinous Xanthomatosis in which a novel acceptor splicing site mutation (IVS6-1G>T) was identified. Family screening revealed the same mutation in his elder brother and the youngest sister. All the three affected siblings were compound heterozygous for IVS6-1G>T and a known missense mutation R372Q (GenBank Accession No. M62401). Significant phenotypic variation was noted among them that the youngest sister was still symptom-free at the time of writing.

Publication types

  • Case Reports

MeSH terms

  • Asian People
  • Cholestanetriol 26-Monooxygenase
  • Female
  • Frameshift Mutation
  • Heterozygote
  • Hong Kong / ethnology
  • Humans
  • Male
  • Middle Aged
  • Mutation, Missense
  • Pedigree
  • Polymorphism, Restriction Fragment Length
  • RNA Splicing*
  • Steroid Hydroxylases / genetics*
  • Xanthomatosis, Cerebrotendinous / genetics*

Substances

  • Steroid Hydroxylases
  • CYP27A1 protein, human
  • Cholestanetriol 26-Monooxygenase