A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.
2016
CRISPR–Cas9-mediated insertion of a naturally occurring benign mutation in blood cell progenitors from patients with sickle cell disease increases fetal hemoglobin expression to levels sufficient to ameliorate the pathological morphology observed in erythrocytes differentiated from these cells.
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