Structural basis of the GM2 gangliosidosis B variant

J Hum Genet. 2003;48(11):582-9. doi: 10.1007/s10038-003-0082-7. Epub 2003 Oct 24.

Abstract

To study the structural basis of the GM2 gangliosidosis B variant, we constructed the three-dimensional structures of the human beta-hexosaminidase alpha-subunit and the heterodimer of the alpha- and beta-subunits, Hex A, by homology modeling. The alpha-subunit is composed of two domains, domains I and II. Nine mutant models due to specific missense mutations were constructed as well and compared with the wild type to determine structural defects. These nine mutations were divided into five groups according to structural defects. R178H is deduced to affect the active site directly, because R178 is important for binding to the substrate. C458Y and W420C are predicted to cause drastic structural changes in the barrel structure carrying the active site pocket. R504C/H is deduced to introduce a disruption of an essential binding with D494 in the beta-subunit for dimerization. R499C/H, located in an extra-helix, is deduced to disrupt hydrogen bonds with domain I and the barrel. R170W and L484P are deduced to affect the interface between domains I and II, causing destabilization. The structural defects reflect the biochemical abnormalities of the disease.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Substitution
  • Cells, Cultured
  • Gangliosidoses, GM2 / enzymology*
  • Gangliosidoses, GM2 / genetics
  • Genetic Variation
  • Hexosaminidase A
  • Humans
  • Isoenzymes / chemistry
  • Isoenzymes / genetics
  • Models, Molecular
  • Mutation
  • Protein Conformation
  • beta-N-Acetylhexosaminidases / chemistry*
  • beta-N-Acetylhexosaminidases / genetics

Substances

  • Isoenzymes
  • Hexosaminidase A
  • beta-N-Acetylhexosaminidases