A gene therapy phase 1 trial in patients with β-thalassemia shows transplantation of autologous CD34+ cells transduced with a lentiviral globin vector after reduced-intensity conditioning achieves long-term engraftment, albeit not transfusion independence, with benign clonal expansions, warranting cautious monitoring of patients.
- Farid Boulad
- Aurelio Maggio
- Michel Sadelain
