Update on fetal hemoglobin gene regulation in hemoglobinopathies

Curr Opin Pediatr. 2011 Feb;23(1):1-8. doi: 10.1097/MOP.0b013e3283420fd0.

Abstract

Purpose of review: The developmental switch from fetal to adult hemoglobin has long fascinated biologists and attracted hematologists given its importance for patients with hemoglobin disorders. New discoveries have reinvigorated the field of globin gene regulation. These results hold promise for improved treatment of the major hemoglobinopathies.

Recent findings: Both genome-wide association studies and traditional linkage studies have identified several genetic loci involved in silencing fetal hemoglobin. BCL11A is a potent silencer of fetal hemoglobin in both mouse and humans. It controls the beta-globin gene cluster in concert with other factors. KLF1, a vital erythroid transcription factor, activates BCL11A and assists in coordinating the switch from fetal to adult hemoglobin. A regulatory network of cell-intrinsic and cell-extrinsic factors maintains the epigenetic homeostasis of the beta-globin cluster and accounts for the precise lineage-specific and developmental stage-specific regulation of the globin genes.

Summary: With an improved understanding of pathways involved in the switch from fetal to adult hemoglobin, new targets have emerged for the treatment of the common hemoglobin disorders, sickle cell anemia and beta-thalassemia.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Carrier Proteins / genetics*
  • Female
  • Fetal Hemoglobin / genetics*
  • Gene Expression Regulation*
  • Genetic Linkage
  • Genome-Wide Association Study
  • Hemoglobinopathies / blood*
  • Hemoglobinopathies / genetics*
  • Humans
  • Kruppel-Like Transcription Factors / genetics*
  • Mice
  • Nuclear Proteins / genetics*
  • Pregnancy
  • Repressor Proteins

Substances

  • BCL11A protein, human
  • Carrier Proteins
  • Kruppel-Like Transcription Factors
  • Nuclear Proteins
  • Repressor Proteins
  • erythroid Kruppel-like factor
  • Fetal Hemoglobin