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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 2. Genetic manipulation of autologous HSCs for TDT gene therapy.

Figure 2.

(A) Patient HSCs are transduced with lentiviral vector (LVV) particles encoding a β-like globin gene. (B) Induction of fetal hemoglobin (HbF, α2γ2) by disrupting the +58 erythroid enhancer in intron 2 of the BCL11A by genome editing nuclease mediated non-homologous end joining (NHEJ) or base editing. (C) Alteration of the γ-globin promoter. Bottom shows disruption of BCL11A or ZBTZ7A repressor binding motifs via genome editing nuclease-mediated NHEJ. Top shows installation of new binding motifs for one of several erythroid transcription factors with adenine base editors.