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1 This ground-breaking study showed that the gene therapy cell product (betibeglogene autotemcel or beti-cel) led to transfusion independence in most treated patients (16/18, 89%), all of whom had genotypes that are associated with severe β-thalassemia (i.e., β 0 /β 0 , β 0 /β IVS1-110 , β IVS1-110 /β IVS1-110 )