A novel silent beta-thalassemia mutation in the distal CACCC box affects the binding and responsiveness to EKLF

Br J Haematol. 2004 Sep;126(6):881-4. doi: 10.1111/j.1365-2141.2004.05146.x.

Abstract

The silent beta-thalassemia mutation, beta(+)-101C-->T, is the only mutation currently described in the distal beta-globin CACCC box. We present a novel mutation, a C-->G transversion, in the same position. Expression analysis in heterozygous subjects demonstrated that the mutation determines a 20% reduction in the output of the beta-globin gene. DNA-protein interaction and transactivation analysis correlated the decrease in the beta-globin synthesis with the reduced binding and transactivation of EKLF to the mutant promoter. These data predict that the beta-101C-->G mutation will display a silent thalassemia phenotype similar to that of the beta-101C-->T mutation.

Publication types

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

MeSH terms

  • DNA-Binding Proteins / metabolism*
  • Female
  • Gene Expression
  • Globins / biosynthesis
  • Globins / genetics*
  • Humans
  • Kruppel-Like Transcription Factors
  • Mutation*
  • Promoter Regions, Genetic / genetics
  • Transcription Factors / metabolism*
  • Transcriptional Activation
  • beta-Thalassemia / genetics*
  • beta-Thalassemia / metabolism

Substances

  • DNA-Binding Proteins
  • Kruppel-Like Transcription Factors
  • Transcription Factors
  • erythroid Kruppel-like factor
  • Globins