Characterization of molecular defects in isovaleryl-CoA dehydrogenase in patients with isovaleric acidemia

Biochemistry. 1998 Jul 14;37(28):10325-35. doi: 10.1021/bi973096r.

Abstract

Isovaleryl-CoA dehydrogenase (IVD) is a homotetrameric mitochondrial flavoenzyme which catalyzes the conversion of isovaleryl-CoA to 3-methylcrotonyl-CoA. PCR of IVD genomic and complementary DNA was used to identify mutations occurring in patients with deficiencies in IVD activity. Western blotting, in vitro mitochondrial import, prokaryotic expression, and kinetic studies of IVD mutants were conducted to characterize the molecular defects caused by the amino acid replacements. Mutations leading to Arg21Pro, Asp40Asn, Ala282Val, Cys328Arg, Val342Ala, Arg363Cys, and Arg382Leu replacements were identified. Western blotting of fibroblast extracts and/or in vitro mitochondrial import experiments indicate that the seven precursor IVD mutant peptides, and a previously identified IVD Leu13Pro mutant, are synthesized and imported into mitochondria. While the IVD Leu13Pro, Arg21Pro, and Cys328Arg mutant peptides are rapidly degraded following mitochondrial import, the other mutant peptides exhibit greater mitochondrial stability, though less than the wild-type enzyme. Active IVD Ala282Val, Val342Ala, Arg363Cys, and Arg382Leu mutants were less stable than wild type when produced in Escherichia coli. The Km values of purified IVD Ala282Val, Val342Ala, and Arg382Leu mutants are 27.0, 2. 8, and 6.9 microM isovaleryl-CoA, respectively, compared to 3.1 microM for the wild type, using the electron-transfer flavoprotein (ETF) fluorescence quenching assay. The catalytic efficiency per mole of FAD content of these three mutants is 4.8, 17.0, and 17.0 microM-1*min-1, respectively, compared to 170 microM-1*min-1 for wild type.

Publication types

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

MeSH terms

  • Amino Acid Metabolism, Inborn Errors / enzymology*
  • Amino Acid Metabolism, Inborn Errors / genetics
  • Animals
  • Cells, Cultured
  • Crystallography, X-Ray
  • DNA Mutational Analysis
  • DNA, Complementary / biosynthesis
  • Enzyme Stability / genetics
  • Escherichia coli / enzymology
  • Escherichia coli / genetics
  • Fibroblasts / chemistry
  • Fibroblasts / enzymology
  • Humans
  • Isovaleryl-CoA Dehydrogenase
  • Kinetics
  • Male
  • Mitochondria, Liver / enzymology
  • Oxidoreductases / chemistry*
  • Oxidoreductases / deficiency
  • Oxidoreductases / genetics*
  • Oxidoreductases / isolation & purification
  • Oxidoreductases Acting on CH-CH Group Donors*
  • Rats
  • Rats, Sprague-Dawley
  • Recombinant Proteins / isolation & purification
  • Recombinant Proteins / metabolism
  • Spectrophotometry

Substances

  • DNA, Complementary
  • Recombinant Proteins
  • Oxidoreductases
  • Oxidoreductases Acting on CH-CH Group Donors
  • Isovaleryl-CoA Dehydrogenase

Associated data

  • PDB/1IVH