Betibeglogene Autotemcel Gene Therapy for Non-beta(0)/beta(0) Genotype beta-Thalassemia

Locatelli, F; Thompson, AA; Kwiatkowski, JL; Porter, JB; Thrasher, AJ; Hongeng, S; Sauer, MG; Thuret, I; Lal, A; Algeri, M; Schneiderman, J; Olson, TS; Carpenter, B; Amrolia, PJ; Anurathapan, U; Schambach, A; Chabannon, C; Schmidt, M; Labik, I; Elliot, H; Guo, RT; Asmal, M; Colvin, RA; Walters, MC

Locatelli, F (通讯作者)，Sapienza Univ Rome, Dept Pediat Hematol & Oncol, IRCCS Osped Pediat Bambino Gesu, Piazza St Onofrio 4, I-00165 Rome, Italy.;Locatelli, F (通讯作者)，Sapienza Univ Rome, Dept Oncol & Cell & Gene Therapy, IRCCS Osped Pediat Bambino Gesu, Piazza St Onofrio 4, I-00165 Rome, Italy.

NEW ENGLAND JOURNAL OF MEDICINE, 2022; 386 (5): 415

Abstract

BACKGROUND Betibeglogene autotemcel (beti-cel) gene therapy for transfusion-dependent beta-thalassemia contains autologous CD34+ hematopoietic stem ce......

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