Clinical features and ETFDH mutation spectrum in a cohort of 90 Chinese patients with late-onset multiple acyl-CoA dehydrogenase deficiency

J Inherit Metab Dis. 2014 May;37(3):399-404. doi: 10.1007/s10545-013-9671-6. Epub 2013 Dec 20.

Abstract

The major cause of lipid storage myopathies (LSM) in China is multiple acyl-CoA dehydrogenase deficiency (MADD) caused by ETFDH mutations. We here present an analysis of the spectrum of ETFDH mutations in the largest cohort of patients with MADD (90 unrelated patients). We identified 61 ETFDH mutations, including 31 novel mutations, which were widely distributed within the coding sequence. Three frequent mutations were identified: c.250G > A (most common in South China), c.770A > G and c.1227A > C (most common in both South and North China). Regional differences of allele frequency and further haplotype analysis suggest the possibility of founder effects of c.250G > A and c.770A > G. These findings promise to provide the basis for implementing a rapid and economical strategy for diagnosing MADD.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Asian People / genetics*
  • Child
  • Child, Preschool
  • Cohort Studies
  • Electron-Transferring Flavoproteins / genetics*
  • Female
  • Haplotypes
  • Humans
  • Iron-Sulfur Proteins / genetics*
  • Male
  • Multiple Acyl Coenzyme A Dehydrogenase Deficiency / genetics*
  • Mutation*
  • Oxidoreductases Acting on CH-NH Group Donors / genetics*

Substances

  • Electron-Transferring Flavoproteins
  • Iron-Sulfur Proteins
  • Oxidoreductases Acting on CH-NH Group Donors
  • electron-transferring-flavoprotein dehydrogenase