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Updated: May 2, 2026
Reprogramming Mouse Embryonic Fibroblasts with Transcription Factors to Induce a Hemogenic Program
Published on: December 16, 2016
Targeted fetal hemoglobin induction for treatment of beta hemoglobinopathies
Susan P Perrine1, Betty S Pace2, Douglas V Faller3
1Hemoglobinopathy-Thalassemia Research Unit, Cancer Center, Department of Medicine, Pediatrics, Pharmacology and Experimental Therapeutics, Boston University School of Medicine, 72 East Concord Street, L-909, Boston, MA 02118, USA.
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.
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