NewslettersHematopoiesis NewsCRISPR–Cas9-Mediated Gene Editing of the BCL11A Enhancer for Pediatric β0/β0 Transfusion-Dependent β-ThalassemiaBy mladbrook - August 4, 20220373The authors report the preliminary results of an ongoing phase I/II trial evaluating safety and efficacy of gene editing therapy in children with blood transfusion-dependent β-thalassemia.[Nature Medicine]Abstract