The mRNA-Binding Protein IGF2BP1 Restores Fetal Hemoglobin in Cultured Erythroid Cells from Patients with β-Hemoglobin Disorders

The mRNA-Binding Protein IGF2BP1 Restores Fetal Hemoglobin in Cultured Erythroid Cells from Patients with β-Hemoglobin Disorders

Sickle cell disease (SCD) and β-thalassemia are caused by structural abnormality or inadequate production of adult hemoglobin (HbA, α2β2), respectively. Individuals with either disorder are asymptomatic before birth because fetal hemoglobin (HbF, α2γ2) is unaffected. Thus, reversal of the switch fro...

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Bibliographic Details
Main Authors: Christopher B. Chambers, Jeffrey Gross, Katherine Pratt, Xiang Guo, Colleen Byrnes, Y. Terry Lee, Donald Lavelle, Ann Dean, Jeffery L. Miller, Andrew Wilber
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
fetal hemoglobin
beta-thalassemia
sickle cell disease
lentivirus
IGF2BP1
hemoglobinopathies
Online Access:http://www.sciencedirect.com/science/article/pii/S232905012030022X

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http://www.sciencedirect.com/science/article/pii/S232905012030022X

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