Fig. 3: Efficient gene marking and robust HbF induction after CD117-ADC conditioning in a rhesus gene therapy model compared to busulfan. | Nature Communications

Fig. 3: Efficient gene marking and robust HbF induction after CD117-ADC conditioning in a rhesus gene therapy model compared to busulfan.

From: Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model

Fig. 3

A Schema of autologous CD34+ cell transplantation with thEpoR and shmiR-BCL11A lentiviral vector transduction (MOI 50). Transplantation occurred either 1 day after myeloablative busulfan conditioning (5.5 mg/kg × 4 days) in rhesus macaques (n = 2), 12U018 and 12U020, or 6 or 10 days after a single injection of 0.3 or 0.4 mg/kg CD117-ADC in rhesus macaques (n = 4) (0.3 mg/kg in ZL13 and ZJ62, 0.4 mg/kg in H635 and H96G), respectively. B Blood counts in rhesus macaques before and after transplantation. C Vector copy number (VCN) in granulocytes and lymphocytes post-transplantation, evaluated by quantitative polymerase chain reaction (qPCR). D HbF-positive red blood cells (F-cells) post-transplantation, evaluated by flow cytometry. E HbF (γ-globin) amounts at the protein level in red blood cells post-transplantation, evaluated by reversed-phase high-performance liquid chromatography (HPLC). pA: a polyadenylation signal, ANKp: an erythroid-specific ankyrin-1 promoter, +51: an erythroid-specific enhancer in the BCL11A gene. Source data are provided as a Source Data file.

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