Mutation of the proton-coupled folate transporter gene (PCFT-SLC46A1) in Turkish siblings with hereditary folate malabsorption

Pediatr Hematol Oncol. 2010 Nov;27(8):614-9. doi: 10.3109/08880018.2010.481705.

Abstract

Hereditary folate malabsorption (HFM) is a rare autosomal recessive disorder characterized by systemic and central nervous system folate deficiency. Turkish siblings are reported with the clinical syndrome of HFM, homozygous for deletion of 2 bases (c.204_205 delCC) within the first exon of the proton-coupled folate transporter (PCFT) gene, causing a frameshift. Low blood and cerebrospinal fluid folate levels were detected at ages 3.5 and 1 month. Treatment with parenteral 5-formyltetrahydrofolate resulted in normal development now at ages 3 and 1 year. Extending current knowledge on the phenotypic manifestations of HFM and the PCFT mutation spectrum will provide opportunities to define possible genotype-phenotype correlations and clarify the basis for the phenotypic variability that is characteristic of this disorder.

Publication types

  • Case Reports

MeSH terms

  • Female
  • Folic Acid / metabolism*
  • Humans
  • Infant
  • Malabsorption Syndromes / genetics*
  • Malabsorption Syndromes / metabolism
  • Male
  • Mutation
  • Proton-Coupled Folate Transporter / genetics*
  • Siblings
  • Turkey

Substances

  • Proton-Coupled Folate Transporter
  • SLC46A1 protein, human
  • Folic Acid