Affordable Access

deepdyve-link
Publisher Website

Targeted Fetal Hemoglobin Induction for Treatment of Beta Hemoglobinopathies

Authors
  • Perrine, Susan P.
  • Pace, Betty S.
  • Faller, Douglas V.1, 2, 3, 4, 5, 6, 2, 4
  • 1 Hemoglobinopathy-Thalassemia Research Unit
  • 2 Cancer Center
  • 3 Department of Medicine, Pediatrics, Pharmacology and Experimental Therapeutics
  • 4 Boston University School of Medicine
  • 5 Department of Pediatrics and Biochemistry and Molecular Biology
  • 6 Georgia Regents University
Type
Published Article
Journal
Hematology/Oncology Clinics of North America
Publisher
Elsevier
Publication Date
Jan 01, 2014
Volume
28
Issue
2
Pages
233–248
Identifiers
DOI: 10.1182/blood-2013-06-506139
Source
Elsevier
Keywords
License
Unknown

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.

Report this publication

Statistics

Seen <100 times