D-3-hydroxyacyl-CoA dehydratase/D-3-hydroxyacyl-CoA dehydrogenase bifunctional protein deficiency: a newly identified peroxisomal disorder

Am J Hum Genet. 1997 Nov;61(5):1153-62. doi: 10.1086/301599.

Abstract

Peroxisomal beta-oxidation proceeds from enoyl-CoA through D-3-hydroxyacyl-CoA to 3-ketoacyl-CoA by the D-3-hydroxyacyl-CoA dehydratase/D-3-hydroxy-acyl-CoA dehydrogenase bifunctional protein (d-bifunctional protein), and the oxidation of bile-acid precursors also has been suggested as being catalyzed by the d-bifunctional protein. Because of the important roles of this protein, we reinvestigated two Japanese patients previously diagnosed as having enoyl-CoA hydratase/L-3-hydroxyacyl-CoA dehydrogenase bifunctional protein (L-bifunctional protein) deficiency, in complementation studies. We found that both the protein and the enzyme activity of the d-bifunctional protein were hardly detectable in these patients but that the active L-bifunctional protein was present. The mRNA level in patient 1 was very low, and, for patient 2, mRNA was of a smaller size. Sequencing analysis of the cDNA revealed a 52-bp deletion in patient 1 and a 237-bp deletion in patient 2. This seems to be the first report of D-bifunctional protein deficiency. Patients previously diagnosed as cases of L-bifunctional protein deficiency probably should be reexamined for a possible d-bifunctional protein deficiency.

Publication types

  • Case Reports

MeSH terms

  • 17-Hydroxysteroid Dehydrogenases*
  • 3-Hydroxyacyl CoA Dehydrogenases / genetics*
  • 3-Hydroxyacyl CoA Dehydrogenases / metabolism
  • Consanguinity
  • Enoyl-CoA Hydratase / metabolism
  • Fatty Acids / metabolism
  • Female
  • Fibroblasts
  • Fluorescent Antibody Technique
  • Humans
  • Hydro-Lyases / deficiency*
  • Hydro-Lyases / genetics*
  • Hydro-Lyases / metabolism
  • Infant
  • Japan
  • Liver / enzymology
  • Microbodies / enzymology*
  • Multienzyme Complexes / deficiency
  • Multienzyme Complexes / genetics*
  • Oxidation-Reduction
  • Palmitates / metabolism
  • Peroxisomal Disorders / enzymology
  • Peroxisomal Disorders / genetics*
  • Peroxisomal Multifunctional Protein-2
  • Polymerase Chain Reaction
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism
  • Sequence Deletion / genetics
  • Stereoisomerism

Substances

  • Fatty Acids
  • Multienzyme Complexes
  • Palmitates
  • RNA, Messenger
  • 17-Hydroxysteroid Dehydrogenases
  • 3-Hydroxyacyl CoA Dehydrogenases
  • Hydro-Lyases
  • Peroxisomal Multifunctional Protein-2
  • HSD17B4 protein, human
  • Enoyl-CoA Hydratase
  • lignoceric acid