NewslettersHematopoiesis News CRISPR–Cas9-Mediated Gene Editing of the BCL11A Enhancer for Pediatric β0/β0 Transfusion-Dependent β-Thalassemia By mladbrook - August 4, 2022 0 The 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