Probing conformational states of glutaryl-CoA dehydrogenase by fragment screening

Acta Crystallogr Sect F Struct Biol Cryst Commun. 2011 Sep 1;67(Pt 9):1060-9. doi: 10.1107/S1744309111014436. Epub 2011 Aug 13.

Abstract

Glutaric acidemia type 1 is an inherited metabolic disorder which can cause macrocephaly, muscular rigidity, spastic paralysis and other progressive movement disorders in humans. The defects in glutaryl-CoA dehydrogenase (GCDH) associated with this disease are thought to increase holoenzyme instability and reduce cofactor binding. Here, the first structural analysis of a GCDH enzyme in the absence of the cofactor flavin adenine dinucleotide (FAD) is reported. The apo structure of GCDH from Burkholderia pseudomallei reveals a loss of secondary structure and increased disorder in the FAD-binding pocket relative to the ternary complex of the highly homologous human GCDH. After conducting a fragment-based screen, four small molecules were identified which bind to GCDH from B. pseudomallei. Complex structures were determined for these fragments, which cause backbone and side-chain perturbations to key active-site residues. Structural insights from this investigation highlight differences from apo GCDH and the utility of small-molecular fragments as chemical probes for capturing alternative conformational states of preformed protein crystals.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Apoenzymes / chemistry
  • Burkholderia pseudomallei / enzymology*
  • Catalytic Domain
  • Crystallography, X-Ray
  • Glutaryl-CoA Dehydrogenase / chemistry*
  • Humans
  • Models, Molecular
  • Phylogeny
  • Protein Structure, Quaternary
  • Structural Homology, Protein

Substances

  • Apoenzymes
  • Glutaryl-CoA Dehydrogenase

Associated data

  • PDB/3D6B
  • PDB/3EOM
  • PDB/3EON
  • PDB/3GNC
  • PDB/3GQT