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. Author manuscript; available in PMC: 2024 Apr 1.
Published in final edited form as: Hematol Oncol Clin North Am. 2023 Apr;37(2):433–447. doi: 10.1016/j.hoc.2022.12.012

Figure 1. Gene therapy for transfusion-dependent β-thalassemia.

Figure 1.

The multi-step process includes: (I) Informed consent, including patient/family education and written consent; (II) mobilization, apheresis collection and enrichment of patient (autologous) hematopoietic stem cells (HSCs); (III) Ex vivo genetic manipulation of HSCs to restore erythroid expression of β-globin or induce fetal hemoglobin (HbF) expression; (IV) Administration of bone marrow conditioning followed by infusion of the modified HSCs.