CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

Short summary

CRISPR-Cas9 gene editing targeting the BCL11A enhancer enabled two patients (one with transfusion-dependent thalassemia, one with sickle cell disease) to achieve transfusion independence and eliminate vaso-occlusive episodes, respectively, more than a year after treatment.

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