The mRNA-Binding Protein IGF2BP1 Restores Fetal Hemoglobin in Cultured Erythroid Cells from Patients with β-Hemoglobin Disorders
Christopher B. Chambers, Jeffrey Gross, Katherine L. Pratt, Xiang Guo, Colleen Byrnes, Y. Terry Lee, Donald Lavelle, Ann Dean, Jeffery L. Miller, Andrew Wilber
Abstract
cells revealed that IGF2BP1-dependent HbF induction could ameliorate the chain imbalance in β-thalassemia or potently suppress expression of sickle β-globin in SCD. In all cases, fetal γ-globin mRNA increased and adult β-globin decreased due, in part, to formation of contacts between the locus control region (LCR) and γ-globin genes. We conclude that expression of IGF2BP1 in adult erythroid cells has the potential to maximize HbF expression in patients with severe β-hemoglobin disorders by reversing the developmental γ- to β-globin switch.
Topics & Concepts
HemoglobinFetal hemoglobinMessenger RNAMolecular biologyBiologyFetusChemistryCell biologyBiochemistryGeneticsGenePregnancyHemoglobinopathies and Related DisordersRNA modifications and cancerBlood groups and transfusion