Diagnosis of fructose-1,6-bisphosphatase deficiency using cultured lymphocyte fraction: a secure and noninvasive alternative to liver biopsy

J Inherit Metab Dis. 2002 Feb;25(1):41-6. doi: 10.1023/a:1015129616599.

Abstract

We report the result of enzymatic and molecular analyses, using cultured lymphocyte fractions (cultivated monocytes), of six Japanese patients (from five families) and one Italian patient with fructose-1,6-bisphosphatase (FBPase) deficiency. Enzymatic analysis demonstrated FBPase deficiency in all seven patients, including the Italian patient whose fructose-1,6-bisphosphatase activity has been reported to be normal in leukocytes but deficient in liver. Molecular analysis of the FBPase gene identified pathogenic mutations in only 8 among the total 12 alleles of six families. We have thus demonstrated the validity of using cultured monocytes as a secure and noninvasive alternative to liver biopsy for accurate diagnosis of FBPase deficiency.

MeSH terms

  • Alleles
  • Cells, Cultured
  • Child
  • Female
  • Fructose-1,6-Diphosphatase Deficiency / diagnosis*
  • Fructose-Bisphosphatase / genetics*
  • Fructose-Bisphosphatase / metabolism
  • Humans
  • Infant
  • Liver / enzymology*
  • Liver / pathology
  • Lymphocytes / cytology
  • Male
  • Monocytes / cytology
  • Monocytes / enzymology*
  • Mutagenesis, Insertional

Substances

  • Fructose-Bisphosphatase