Defective succinic semialdehyde dehydrogenase activity in 4-hydroxybutyric aciduria

Eur J Pediatr. 1984 Sep;142(4):257-9. doi: 10.1007/BF00540247.

Abstract

Succinic semialdehyde dehydrogenase deficiency has been demonstrated in a fourth patient with 4-hydroxybutyric aciduria. Lysates of freshly isolated lymphocytes and cultured lymphoblasts of the patient had much lower than control activity in the conversion of U-14C-4-aminobutyric acid to 14C-succinic acid in an assay designed to estimate succinic semialdehyde dehydrogenase utilizing endogenous 4-aminobutyrate transaminase. Lymphocyte and lymphoblast lysates of the patient accumulated U-14C-succinic semialdehyde when incubated with U-14C-4-aminobutyric acid and NAD+ whereas none could be detected in controls. Assays using U-14C-succinic semialdehyde as substrate for succinic semialdehyde dehydrogenase in lysates of cultured lymphoblasts characterized the patient as having a severe deficiency of succinic semialdehyde dehydrogenase. The data indicate that defective activity of succinic semialdehyde dehydrogenase is responsible for 4-hydroxybutyric aciduria.

Publication types

  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • 4-Aminobutyrate Transaminase / blood
  • Adult
  • Carboxy-Lyases / blood
  • Humans
  • Hydroxybutyrate Dehydrogenase / deficiency*
  • Hydroxybutyrates / urine*
  • Kinetics
  • Lymphocytes / enzymology
  • Methylmalonyl-CoA Decarboxylase
  • Sodium Oxybate / urine*

Substances

  • Hydroxybutyrates
  • Sodium Oxybate
  • Hydroxybutyrate Dehydrogenase
  • 4-hydroxybutyrate dehydrogenase
  • 4-Aminobutyrate Transaminase
  • Carboxy-Lyases
  • Methylmalonyl-CoA Decarboxylase