Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia.

Kwiatkowski JL, Thompson AA, Schneiderman J, Thuret I, Kulozik AE, Yannaki E, Cavazzana M, Hongeng S, Olson TS, Sauer MG, Thrasher AJ, Lal A, Rasko JE, Kunz JB, Kinney MA, Chawla A, Ali S, Tao G, Thakar H, Paramore C, Witthuhn N, Walters MC, Locatelli F