New SUCLG1 patients expanding the phenotypic spectrum of this rare cause of mild methylmalonic aciduria

Mitochondrion. 2010 Jun;10(4):335-41. doi: 10.1016/j.mito.2010.02.006. Epub 2010 Mar 1.

Abstract

Deficiencies in two subunits of the succinyl-coenzyme A synthetase (SCS) have been involved in patients with encephalomyopathy and mild methylmalonic aciduria (MMA). In this study, we described three new SUCLG1 patients and performed a meta-analysis of the literature. Our report enlarges the phenotypic spectrum of SUCLG1 mutations and confirms that a characteristic metabolic profile (presence of MMA and C4-DC carnitine in urines) and basal ganglia MRI lesions are the hallmarks of SCS defects. As mitochondrial DNA depletion in muscle is not a constant finding in SUCLG1 patients, this may suggest that diagnosis should not be based on it, but also that alternative physiopathological mechanisms may be considered to explain the combined respiratory chain deficiency observed in SCS patients.

Publication types

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

MeSH terms

  • Adolescent
  • Animals
  • Basal Ganglia / diagnostic imaging
  • Basal Ganglia / pathology
  • Carnitine / analogs & derivatives
  • Carnitine / urine
  • Child
  • Child, Preschool
  • Female
  • Genetic Diseases, Inborn / diagnosis*
  • Genetic Diseases, Inborn / pathology*
  • Humans
  • Infant
  • Infant, Newborn
  • Magnetic Resonance Imaging
  • Male
  • Methylmalonic Acid / urine
  • Mitochondrial Proteins / deficiency*
  • Models, Molecular
  • Protein Structure, Tertiary
  • Radiography
  • Succinate-CoA Ligases / deficiency*

Substances

  • Mitochondrial Proteins
  • Methylmalonic Acid
  • Succinate-CoA Ligases
  • Carnitine