Monolysocardiolipin in cultured fibroblasts is a sensitive and specific marker for Barth Syndrome

J Lipid Res. 2006 Oct;47(10):2346-51. doi: 10.1194/jlr.D600024-JLR200. Epub 2006 Jul 27.

Abstract

Barth Syndrome (BTHS) is an X-linked recessive disorder that results in abnormal metabolism of the mitochondrial phospholipid cardiolipin (CL). CLs are decreased and monolysocardiolipins (MLCLs), intermediates in CL metabolism, are increased in a variety of tissues. Measurement of decreased CL levels in skin fibroblasts has previously been proposed as a diagnostic test for BTHS. We investigated whether elevated MLCL is specific for BTHS and whether the MLCL-to-CL ratio is a more sensitive and specific marker for BTHS. We measured CLs and MLCLs in skin fibroblasts from 5 BTHS patients, 8 controls, and 14 patients with biochemical and clinical findings similar to those in BTHS (group D), using high performance liquid chromatography-mass spectrometry. Our results showed a clear decrease of CL in combination with a marked increase of MLCL in fibroblasts from BTHS patients when compared with controls. MLCL/CL ratios ranged from 0.03-0.12 in control fibroblasts and from 5.41-13.83 in BTHS fibroblasts. In group D, the MLCL/CL ratio range was 0.02-0.06. We therefore conclude that elevations of MLCLs are specific for BTHS and that the MLCL/CL ratio in fibroblasts is a better diagnostic marker than CL alone. We also report the finding of two novel mutations in the TAZ gene that cause BTHS.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biomarkers / analysis
  • Cardiolipins / analysis*
  • Cell Line
  • Child, Preschool
  • Chromosomes, Human, X
  • Fibroblasts / chemistry*
  • Fibroblasts / cytology
  • Genetic Diseases, X-Linked / diagnosis*
  • Genetic Diseases, X-Linked / metabolism*
  • Growth Disorders / diagnosis
  • Growth Disorders / metabolism
  • Humans
  • Infant
  • Infant, Newborn
  • Lysophospholipids / analysis*
  • Muscles / metabolism
  • Mutation
  • Syndrome

Substances

  • Biomarkers
  • Cardiolipins
  • Lysophospholipids
  • monolysocardiolipin