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Targeted fetal hemoglobin induction for treatment of beta hemoglobinopathies.
- 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.)
- Subjects :
- Anemia, Sickle Cell drug therapy
Anemia, Sickle Cell genetics
Animals
Butyrates pharmacology
Butyrates therapeutic use
Hemoglobinopathies drug therapy
Humans
Models, Genetic
Transcriptional Activation drug effects
alpha-Globins genetics
beta-Thalassemia drug therapy
beta-Thalassemia genetics
Fetal Hemoglobin genetics
Hemoglobinopathies genetics
Transcriptional Activation genetics
Subjects
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