Genetic testing of Korean familial hypercholesterolemia using whole-exome sequencing

PLoS One. 2015 May 11;10(5):e0126706. doi: 10.1371/journal.pone.0126706. eCollection 2015.

Abstract

Familial hypercholesterolemia (FH) is a genetic disorder with an increased risk of early-onset coronary artery disease. Although some clinically diagnosed FH cases are caused by mutations in LDLR, APOB, or PCSK9, mutation detection rates and profiles can vary across ethnic groups. In this study, we aimed to provide insight into the spectrum of FH-causing mutations in Koreans. Among 136 patients referred for FH, 69 who met Simon Broome criteria with definite family history were enrolled. By whole-exome sequencing (WES) analysis, we confirmed that the 3 known FH-related genes accounted for genetic causes in 23 patients (33.3%). A substantial portion of the mutations (19 of 23 patients, 82.6%) resulted from 17 mutations and 2 copy number deletions in LDLR gene. Two mutations each in the APOB and PCSK9 genes were verified. Of these anomalies, two frameshift deletions in LDLR and one mutation in PCSK9 were identified as novel causative mutations. In particular, one novel mutation and copy number deletion were validated by co-segregation in their relatives. This study confirmed the utility of genetic diagnosis of FH through WES.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Apolipoproteins B / genetics
  • Asian People
  • Female
  • Genetic Testing / methods*
  • Humans
  • Hypercholesterolemia / diagnosis*
  • Hypercholesterolemia / genetics*
  • Male
  • Middle Aged
  • Mutation
  • Proprotein Convertase 9
  • Proprotein Convertases / genetics
  • Receptors, LDL / genetics
  • Serine Endopeptidases / genetics

Substances

  • Apolipoproteins B
  • LDLR protein, human
  • Receptors, LDL
  • PCSK9 protein, human
  • Proprotein Convertase 9
  • Proprotein Convertases
  • Serine Endopeptidases

Grants and funding

Financial support was provided by the Basic Science Research Program through the National Research Foundation of Korea, which was funded by the Ministry of Education, Science, and Technology (2012R1A1A2039828, 2012R1A4A1029061, SH Lee). Funding was also provided by the Creative Allied Project (Korean Research Council of Fundamental Science and Technology, SH Lee) and Korea Health Technology R&D Project (Korea Health Industry Development Institute, Ministry of Health & Welfare; HI14C0070, JH Lee; HI13C2163, D Bang).