Evaluation of the biotinidase activity in hepatic glycogen storage disease patients. Undescribed genetic finding associated with atypical enzymatic behavior: an outlook

J Inherit Metab Dis. 2010 Oct;33(Suppl 2):S289-94. doi: 10.1007/s10545-010-9139-x. Epub 2010 Jun 8.

Abstract

Repeated evaluation of biotinidase (BTD) activity was carried out for a long-term follow-up in patients with hepatic glycogen storage diseases (GSDs). The results indicated inter-intra variability among the GSD-Ia, GSD-III and GSD-IX patients. In addition, a c.1330G>C transversion in the BTD gene, resulting in a p.Asp444His substitution was detected in one allele of a GSD-Ia patient with sustained normal enzyme activity. Thus far, it is necessary to be cautious in the interpretation of the results of BTD activity as a presumptive GSD diagnostic element. It is not known why plasma BTD activity increases in GSDs patients, or the clinical importance of the increment. When viewed from a global perspective, there are some lines of biotin biology that could indicate a relationship between BTD´s behavior and GSDs.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Argentina
  • Biomarkers / blood
  • Biotinidase / blood*
  • Biotinidase / genetics
  • Case-Control Studies
  • DNA Mutational Analysis
  • Genotype
  • Glycogen Storage Disease / blood
  • Glycogen Storage Disease / diagnosis
  • Glycogen Storage Disease / enzymology*
  • Glycogen Storage Disease / genetics
  • Glycogen Storage Disease Type I / enzymology
  • Glycogen Storage Disease Type III / enzymology
  • Humans
  • Liver / enzymology*
  • Mutation
  • Phenotype
  • Up-Regulation

Substances

  • Biomarkers
  • Biotinidase

Supplementary concepts

  • Hepatorenal form of glycogen storage disease

Associated data

  • RefSeq/NM_000060