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CRISPR–Cas9-mediated gene editing of the BCL11A enhancer for pediatric β0/β0 transfusion-dependent β-thalassemia

Gene TherapiesGeneticsHematologyGenetic EngineeringGene EditingGenome EngineeringGenome EditingCrispr–cas9-mediated Gene EditingBcl11a EnhancerMedicineCell BiologyCrispr