Thalassaemia-like carriers not linked to the beta-globin gene cluster

Br J Haematol. 2006 Mar;132(5):640-50. doi: 10.1111/j.1365-2141.2005.05915.x.

Abstract

This study describes the largest series reported to date, of individuals belonging to unrelated families carrying a beta-thalassaemia-like phenotype in whom the beta-globin gene was found to be structurally intact by sequence analysis. This genetic determinant appears haematologically heterogeneous, displaying either a silent beta-thalassaemia-like phenotype or a typical beta-thalassaemia carrier-like phenotype in different families. Compound heterozygosity for both beta-thalassaemia-like determinant and typical beta-thalassaemia allele resulted either in thalassaemia intermedia or thalassaemia major. By linkage analysis both the silent and the typical beta-like determinants were found not to be linked to the beta-globin cluster. Sequence analysis of the hypersensitive site cores of locus control region and of the genes coding for the transcription factors erythroid Kruppel-like factor and nuclear factor (erythroid-derived 2) were normal. beta-globin mRNA levels determined by real-time polymerase chain reaction were reduced in both types of beta-like carriers. These results indicate the existence of causative genetic determinants not yet molecularly defined, but most likely, resulting from either the reduction or loss of function of a gene coding for unknown transcriptional regulator(s) of the beta-globin gene. The knowledge of these rare beta-thalassaemia-like determinants have implications for clinical and, especially, prenatal diagnosis of beta-thalassaemia.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alleles
  • Blood Transfusion
  • Carrier State
  • Female
  • Genetic Linkage
  • Globins / genetics*
  • Haplotypes
  • Humans
  • Italy
  • Locus Control Region
  • Male
  • Multigene Family
  • Pregnancy
  • RNA, Messenger / analysis
  • Reverse Transcriptase Polymerase Chain Reaction
  • Sequence Analysis, DNA
  • Thalassemia / genetics*
  • Thalassemia / therapy

Substances

  • RNA, Messenger
  • Globins