Use of fluorescent substrates for characterization of Gaucher disease mutations

Blood Cells Mol Dis. 2005 Jul-Aug;35(1):57-65. doi: 10.1016/j.bcmd.2005.03.006.

Abstract

Gaucher disease results from impaired activity of the lysosomal enzyme beta-glucocerebrosidase. More than 200 mutations within the glucocerebrosidase gene have been associated with this disease. In this study we tested the effect of several mutations (K157Q, D140H, E326K, D140H+E326K, V394L and R463C) on RNA stability, protein stability and activity toward four different fluorescent substrates (LR-12-GC, Bodipy-12-GC, LR-0-PAP-glucose and 4-MUG), using the vaccinia-derived expression system. The results indicated that the K157Q mutation leads to RNA instability, causing low protein levels and a concomitant reduction in beta-glucocerebrosidase activity. All other tested mutations led to production of glucocerebrosidase RNA and protein with stabilities comparable to those of the normal counterpart. The D140H variant exhibited a high activity toward the tested substrates while the variant enzymes containing either the E326K or D140H and E326k mutations together expressed low beta-glucocerebrosidase activity. The V394L variant exhibited low activity toward the tested substrates, while a higher activity was presented by the R463C containing glucocerebrosidase variant. Our results strongly indicated that the LR-12-GC substrate distinguishes between severities of different mutant glucocerebrosidase variants overexpressed in a heterologous system.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Line
  • Fluorescent Dyes
  • Gaucher Disease / diagnosis
  • Gaucher Disease / genetics*
  • Genetic Predisposition to Disease
  • Glucosylceramidase / genetics
  • Glucosylceramidase / metabolism*
  • Humans
  • Mutation*
  • Predictive Value of Tests
  • RNA Stability / genetics
  • Severity of Illness Index*
  • Substrate Specificity
  • Transfection

Substances

  • Fluorescent Dyes
  • Glucosylceramidase

Associated data

  • OMIM/230800
  • OMIM/231000