Identification of LDLR mutations in two Chinese pedigrees with familial hypercholesterolemia

J Pediatr Endocrinol Metab. 2012;25(7-8):769-73. doi: 10.1515/jpem-2012-0024.

Abstract

Familial hypercholesterolemia (FH) is an autosomal dominant, inherited disease (OMIM 143890) characterized by elevated serum cholesterol bound to low-density lipoprotein (LDL). It is mainly caused by mutations of the low-density lipoprotein receptor gene (LDLR). In this study, we investigated two Chinese pedigrees with FH. The probands were a 9-year-old boy and a 1-year-old boy, who had high LDL-C levels. The proband in family A showed skin xanthoma. We sequenced the promoter and all exons and exon-intron boundaries of the LDLR gene to detect potential mutations. Compound heterozygote of c.1747C>T and c.2054C>T was detected in the proband of family A, and a heterozygous indel mutation c.551_553 delGTAinsTT was discovered in the second family. The c.1747C>T and c.2054C>T mutations, which have been reported previously, result in His583Tyr and Pro685Leu substitutions, respectively. The novel c.551_553 delGTAinsTT indel mutation causes a frameshift, which results in a p.Cys184Phe fs21X mutation in the corresponding protein.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Asian People / genetics
  • Base Sequence
  • Child
  • DNA Mutational Analysis
  • Family
  • Humans
  • Hyperlipoproteinemia Type II / ethnology
  • Hyperlipoproteinemia Type II / genetics*
  • Infant
  • Male
  • Mutation, Missense* / physiology
  • Pedigree
  • Receptors, LDL / genetics*

Substances

  • LDLR protein, human
  • Receptors, LDL