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Initial Results from the BEACON Clinical Study: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises

Ashish O. Gupta,Akshay Sharma,Haydar Frangoul,Jignesh Dalal,Julie Kanter,Asif Alavi,John Dipersio,Mary Eapen,Jennifer J. Jaroscak,Ernesto Ayala, Edward D. Ziga,Stacey Rifkin-Zenenberg, Alex C. Minella, Guo Chen, Yinzhong Chen, Priya S. Chockalingam, Ling Lin,Marcelyne Joseney-Antoine, Leanne Ianniello, Beth Gardner,Adam J. Hartigan,Giuseppe Ciaramella,Sunita Goyal, Amy Simon,Alexis Thompson,Matthew M. Heeney

BLOOD（2024）

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