Abstract
The clinical severity and lifelong morbidity of sickle cell disease (SCD) and b-thalassemia, the major disorders of b-globin gene mutations, can be ameliorated by production of fetal hemoglobin (HbF). In fetal life, g-globin (encoded by 2 duplicated genes in the b-globin locus) does not use the mutated adult b-hemoglobin (HbA). The HbF to HbA transition is regulated by a repressor, Bcl11a, which silences fetal g-globin. Given our overarching goal to develop a fetal gene therapy for SCD which “unsilences” g-globin, we aimed to disrupt the Bcl11a erythroid-specific enhancer region using three different synthetic single guide RNA (sgRNA) sequences with CRISPR/Cas9 in murine erythroleukemia (MEL) cells (Fig.
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