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Targeted fetal hemoglobin induction for treatment of beta hemoglobinopathies.

Authors :
Perrine SP
Pace BS
Faller DV
Source :
Hematology/oncology clinics of North America [Hematol Oncol Clin North Am] 2014 Apr; Vol. 28 (2), pp. 233-48.
Publication Year :
2014

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.<br /> (Copyright © 2014 Elsevier Inc. All rights reserved.)

Details

Language :
English
ISSN :
1558-1977
Volume :
28
Issue :
2
Database :
MEDLINE
Journal :
Hematology/oncology clinics of North America
Publication Type :
Academic Journal
Accession number :
24589264
Full Text :
https://doi.org/10.1016/j.hoc.2013.11.009