High cerebral guanidinoacetate and variable creatine concentrations in argininosuccinate synthetase and lyase deficiency: implications for treatment?

Mol Genet Metab. 2006 Nov;89(3):274-6. doi: 10.1016/j.ymgme.2006.02.005. Epub 2006 Apr 3.

Abstract

Cerebral creatine and guanidinoacetate and blood and urine metabolites were studied in four patients with argininosuccinate synthetase (ASS) or argininosuccinate lyase (ASL) deficiency receiving large doses of arginine. Urine and blood metabolites varied largely. Cerebral guanidinoacetate was increased in all patients, while cerebral creatine was low in ASS and high in ASL deficiency. Because high cerebral guanidinoacetate might be toxic, lowering the arginine supplementation with additional creatine supplementation might be important.

MeSH terms

  • Amino Acid Metabolism, Inborn Errors / therapy*
  • Arginine / blood
  • Argininosuccinate Synthase / deficiency*
  • Argininosuccinic Aciduria*
  • Brain / metabolism*
  • Child
  • Child, Preschool
  • Creatine / blood
  • Creatine / metabolism*
  • Creatine / urine
  • Female
  • Glycine / analogs & derivatives*
  • Glycine / blood
  • Glycine / metabolism
  • Glycine / urine
  • Humans
  • Infant, Newborn
  • Male
  • Pregnancy

Substances

  • Arginine
  • Argininosuccinate Synthase
  • glycocyamine
  • Creatine
  • Glycine