A novel mutation of late-onset very-long-chain acyl-CoA dehydrogenase deficiency

Pediatr Neurol. 2002 Aug;27(2):136-7. doi: 10.1016/s0887-8994(02)00404-6.

Abstract

We describe a novel mutation in three patients with the myopathic form of very-long-chain acyl-CoA dehydrogenase deficiency. Three siblings born to second-degree cousins of Jewish-Iraqi origin exhibited rhabdomyolysis and myoglobinuria as the presenting signs of the mild late-onset form of very-long-chain acyl-CoA dehydrogenase deficiency. We screened the patients for mutations in the very-long-chain acyl-CoA dehydrogenase gene by amplification of all 20 exons and analysis by single-stranded conformation variance on gel electrophoresis. The patients were homozygous for a novel mutation G637A that alters alanine 173 to threonine. We hypothesize that this missense substitution caused a mild change of enzyme function correlating with the mild clinical features and, thus, favoring a genotype-phenotype correlation.

Publication types

  • Case Reports

MeSH terms

  • Acyl-CoA Dehydrogenase, Long-Chain
  • Acyl-CoA Dehydrogenases / deficiency*
  • Acyl-CoA Dehydrogenases / genetics*
  • Adolescent
  • Alanine Dehydrogenase
  • Alcohol Oxidoreductases / genetics
  • Amino Acid Oxidoreductases / genetics
  • DNA Mutational Analysis
  • Diseases in Twins / genetics
  • Electrophoresis, Agar Gel / methods
  • Exons
  • Female
  • Humans
  • Metabolism, Inborn Errors / diagnosis
  • Metabolism, Inborn Errors / genetics*
  • Mutation, Missense / genetics*
  • Nucleic Acid Amplification Techniques

Substances

  • Alcohol Oxidoreductases
  • L-threonine 3-dehydrogenase
  • Acyl-CoA Dehydrogenases
  • Acyl-CoA Dehydrogenase, Long-Chain
  • Amino Acid Oxidoreductases
  • Alanine Dehydrogenase