Generation Of New Cell Models To Screen Improved Vectors For Functional Hemoglobin Expression
BLOOD(2017)
摘要
Gene addition of a functional copy of β-globin and reactivation of fetal hemoglobin (HbF) are promising therapeutical approaches for β-globinopathies such as Sickle Cell Disease (SCD) and β-thalassemia (β-thal).
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