A novel mechanism for thalassaemia intermedia

Lancet. 2002 Jan 12;359(9301):132-3. doi: 10.1016/s0140-6736(02)07338-5.

Abstract

Thalassaemia intermedia is a moderate form of thalassaemia resulting from various genetic defects. We report an undescribed mechanism leading to this condition: a somatic deletion of the beta-globin gene in the haemopoietic lineage of a heterozygous beta-thalassaemic patient. We did molecular studies and haemoglobin analysis of the patient and his parents. We found that the deletion gives rise to a mosaic of cells with either one or no functional beta-globin gene and it extends to a region of frequent loss of heterozygosity called LOH11A, which is located close to the beta-globin locus. Thus, loss of heterozygosity can be a cause of non-malignant genetic disease.

Publication types

  • Case Reports
  • Letter

MeSH terms

  • Anemia / genetics
  • Child
  • Codon, Nonsense / genetics
  • Gene Deletion*
  • Genes, Dominant / genetics
  • Genetic Testing
  • Genotype
  • Globins / genetics*
  • Growth Disorders / genetics
  • Hemoglobins / analysis
  • Hepatomegaly / genetics
  • Heterozygote
  • Humans
  • In Situ Hybridization, Fluorescence
  • Loss of Heterozygosity / genetics*
  • Male
  • Pedigree
  • Restriction Mapping
  • Severity of Illness Index
  • Splenomegaly / genetics
  • beta-Thalassemia / blood
  • beta-Thalassemia / classification
  • beta-Thalassemia / complications
  • beta-Thalassemia / genetics*

Substances

  • Codon, Nonsense
  • Hemoglobins
  • Globins