Response of Patients with Transfusion-Dependent ß-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for ß-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers.

Mark C. Walters, David H.K. Chui, John J Farrell, Ashutosh Lal, Franco Locatelli, Janet L. Kwiatkowski, John B. Porter, Martin G. Sauer, Isabelle Thuret, Suradej Hongeng, Andreas E. Kulozik, Adrian J. Thrasher, Evangelia Yannaki, Julia Yang, Dustin Whitney, Alexandria Petrusich, Richard A. Colvin, Alexis A. Thompson