Targeted fetal hemoglobin induction for treatment of beta hemoglobinopathies

Susan P. Perrine, Betty Sue Pace, Douglas V. Faller

Research output: Contribution to journalReview article

28 Scopus citations

Abstract

Fetal globin (gamma globin; HBG) is normally expressed during fetal life and prevents the clinical manifestations of beta hemoglobinopathies before birth. HBG genes are normally integrated in hematopoietic stem cells in all humans, and are at least partially amenable to reactivation. Inducing expression of fetal globin (HBG) gene expression to 60% to 70% of alpha globin synthesis produces a β-thalassemia trait phenotype, and reduces anemia. Tailoring combinations of therapeutics to patient subsets characterized for quantitative trait loci which modulate basal fetal hemoglobin and erythroid cell survival should provide effective amelioration of clinical symptoms in β-thalassemia and sickle cell disease.

Original languageEnglish (US)
Pages (from-to)233-248
Number of pages16
JournalHematology/Oncology Clinics of North America
Volume28
Issue number2
DOIs
Publication statusPublished - Apr 1 2014

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Keywords

  • Beta-thalassemia
  • Fetal hemoglobin
  • Genetic modifiers
  • Sickle cell disease
  • Stress signaling

ASJC Scopus subject areas

  • Oncology
  • Hematology
  • Medicine(all)

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