Gene Therapy for β-Thalassemia Of 23 patients with transf | UPDATES IN MEDICINE
Gene Therapy for β-Thalassemia
Of 23 patients with transfusion-dependent β-thalassemia who received gene-transfected autologous hematopoietic cells, 91% became transfusion independent; the remaining 2 patients had a low level of engraftment of vector-expressing cells. With a median of 29.5 months of follow-up, the average hemoglobin level was 11.7 g per deciliter.
