Respiratory chain deficiency in a female with Aicardi-Goutières syndrome

Dev Med Child Neurol. 2006 Mar;48(3):227-30. doi: 10.1017/S001216220600048X.

Abstract

Aicardi-Goutières syndrome (AGS) is an early-onset progressive encephalopathy characterized by calcifications of the basal ganglia, white matter abnormalities, chronic cerebrospinal fluid (CSF) lymphocytosis, and/or a raised level of CSF interferon (INF)-alpha. We report a female with mitochondrial respiratory chain deficiency fulfilling the criteria of AGS. Disease onset was in the first year of age with seizures and psychomotor regression. To date, at 4 years of age, she presents a severe encephalopathy, increased INF-alpha in the CSF, and calcifications of basal ganglia on computerized tomography. Cerebral magnetic resonance imaging showed bilateral and symmetric hypersignal of the posterior white matter. A complex I deficiency of the mitochondrial respiratory chain was found in skeletal muscle, which was associated with a complex IV deficiency in cultured skin fibroblasts. The question of whether this oxidative phosphorylation deficiency is primary or secondary in AGS is open to debate. We suggest giving consideration to systematic evaluation of the mitochondrial respiratory chain in skeletal muscle and skin fibroblasts of other AGS patients.

Publication types

  • Case Reports

MeSH terms

  • Basal Ganglia / pathology*
  • Calcinosis / complications*
  • Calcinosis / pathology*
  • Chromatography, Gas
  • Dementia, Vascular / complications*
  • Dementia, Vascular / pathology*
  • Female
  • Humans
  • Interferon-alpha / cerebrospinal fluid*
  • Ketone Bodies / metabolism
  • Lactic Acid / metabolism
  • Lymphocytosis / cerebrospinal fluid*
  • Lymphocytosis / complications*
  • Magnetic Resonance Imaging
  • Mitochondrial Diseases / complications*
  • Mitochondrial Diseases / pathology
  • Muscle, Skeletal / metabolism
  • Muscle, Skeletal / pathology
  • Psychomotor Disorders / complications
  • Pyruvic Acid / metabolism
  • Seizures / complications
  • Syndrome

Substances

  • Interferon-alpha
  • Ketone Bodies
  • Lactic Acid
  • Pyruvic Acid