A novel type of congenital hypochromic anemia associated with a nonsense mutation in the STEAP3/TSAP6 gene

Blood. 2011 Dec 15;118(25):6660-6. doi: 10.1182/blood-2011-01-329011. Epub 2011 Oct 26.

Abstract

STEAP3/TSAP6 encodes a ferrireductase that is involved in the acquisition of iron by developing erythroblasts and steap3/tsap6 null-mice display severe microcytic anemia. We report a family in which 3 siblings born to healthy parents display transfusion-dependent hypochromic anemia. A nonsense STEAP3/TSAP6 was identified in the siblings at the heterozygous state. This mutation was inherited from their father while no mutation was found in their mother. A large variability of expression was found between normal alleles in a control population, confirming a previous report that STEAP3/TSAPS6 is an expressed quantitative trait locus (e-QTL). Determination of the relative allele expression showed that the "normal" allele was expressed at a significantly higher level in the father than in the affected siblings relative to the shared mutated allele. The blood level of STEAP3/TSAP6 mRNA was severely reduced in the siblings, while both parents were in the lower range of normal controls. The STEAP3/TSAP6 protein was also reduced in lymphocytic cell lines from the patients. Collectively, our data support the hypothesis that STEAP3/TSAP6 deficiency leads to severe anemia in the affected siblings and results from the combination of a mutated allele inherited from their father and a weakly expressed allele inherited from their mother.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Anemia, Hypochromic / blood
  • Anemia, Hypochromic / congenital
  • Anemia, Hypochromic / genetics*
  • Animals
  • Blotting, Western
  • Cell Cycle Proteins
  • Cell Line, Transformed
  • Cells, Cultured
  • Child
  • Codon, Nonsense*
  • DNA Mutational Analysis
  • Embryo, Mammalian / cytology
  • Embryo, Mammalian / metabolism
  • Family Health
  • Female
  • Fibroblasts / cytology
  • Fibroblasts / metabolism
  • Gene Expression
  • Humans
  • Male
  • Membrane Proteins / genetics
  • Membrane Proteins / metabolism
  • Mice
  • Mice, Knockout
  • Oncogene Proteins / genetics*
  • Oncogene Proteins / metabolism
  • Oxidoreductases
  • Pedigree
  • RNA, Messenger / blood
  • RNA, Messenger / genetics
  • Reverse Transcriptase Polymerase Chain Reaction
  • Young Adult

Substances

  • Cell Cycle Proteins
  • Codon, Nonsense
  • Membrane Proteins
  • Oncogene Proteins
  • RNA, Messenger
  • Oxidoreductases
  • STEAP3 protein, human
  • TSAP6 protein, mouse