Hereditary spherocytosis with spectrin deficiency related to null mutations of the beta-spectrin gene

Blood Cells Mol Dis. 1998 Jun;24(2):251-61. doi: 10.1006/bcmd.1998.0190.

Abstract

Spectrin deficiency is the most common deficiency found in HS. It is heterogeneous in terms of clinical expression, inheritance (dominant or recessive) and underlying genetic defects (related to alpha- or beta-spectrin gene defects or secondary to ankyrin gene defects). We studied a sampling of French dominant HS families, selected after linkage analyses, and found the presence of mutations resulting in the silencing of the mutant beta-spectrin allele. In three HS families, one haploid set of beta-spectrin mRNA was undectectable. In two families, a deletion of 8 bases (leading to a frameshift and a premature stop codon) and a nonsense mutation were identified, respectively. In the third HS family, we were unable to characterize a relevant mutation but the loss of heterozygosity at the cDNA level suggested the presence of a null mutation of the beta-spectrin allele. Sequencing of the beta-spectrin gene has also uncovered several new polymorphisms in the coding region of the beta-spectrin gene which will be very useful for detecting loss of heterozygosity at the cDNA level and designating the beta-spectrin gene as the culprit one.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Substitution
  • Anion Exchange Protein 1, Erythrocyte / analysis
  • Ankyrins / blood
  • DNA Mutational Analysis
  • DNA, Complementary / genetics
  • Female
  • France / epidemiology
  • Genes, Dominant
  • Genetic Heterogeneity
  • Humans
  • Loss of Heterozygosity
  • Male
  • Pedigree
  • Polymorphism, Genetic
  • RNA, Messenger / blood
  • Spectrin / deficiency
  • Spectrin / genetics*
  • Spherocytosis, Hereditary / blood
  • Spherocytosis, Hereditary / epidemiology
  • Spherocytosis, Hereditary / genetics*

Substances

  • Anion Exchange Protein 1, Erythrocyte
  • Ankyrins
  • DNA, Complementary
  • RNA, Messenger
  • Spectrin