Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial

• Main Authors: Farid Boulad, Aurelio Maggio, Xiuyan Wang, Paolo Moi, Santina Acuto, Friederike Kogel, Chayamon Takpradit, Susan Prockop, Jorge Mansilla-Soto, Annalisa Cabriolu, Ashlesha Odak, Jinrong Qu, Keyur Thummar, Fang Du, Lingbo Shen, Simona Raso, Rita Barone, Rosario Di Maggio, Lorella Pitrolo, Antonino Giambona, Maura Mingoia, John K. Everett, Pascha Hokama, Aoife M. Roche, Vito Adrian Cantu, Hriju Adhikari, Shantan Reddy, Eric Bouhassira, Narla Mohandas, Frederic D. Bushman, Isabelle Rivière, Michel Sadelain
• Other Authors: Siriraj Hospital
• Format: Article
• Published: 2022
• Subjects: Biochemistry, Genetics and Molecular Biology
• Online Access: https://repository.li.mahidol.ac.th/handle/123456789/73535
• Institution: Mahidol University

β-Thalassemias are inherited anemias that are caused by the absent or insufficient production of the β chain of hemoglobin. Here we report 6–8-year follow-up of four adult patients with transfusion-dependent β-thalassemia who were infused with autologous CD34+ cells transduced with the TNS9.3.55 len...