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. Author manuscript; available in PMC: 2020 Mar 1.
Published in final edited form as: J Allergy Clin Immunol. 2018 Sep 5;143(3):852–863. doi: 10.1016/j.jaci.2018.08.024

Figure 2: Scheme of gamma-retrovirus and lentivirus based gene therapy with busulfan and discontinuation of enzyme replacement therapy.

Figure 2:

After obtaining consent of patients/guardians for autologous hematopoietic stem cell (HSC) gene therapy, patients are screened and admitted for bone marrow (BM) harvest and conditioning with low dose busulfan, with adjustment in accordance to pharmacokinetics (pK) predetermined targets. CD34+ HSC are isolated, transduced with gamma retroviral vector (A) or lentiviral vector (B) containing the ADA gene, and reinfused through a central venous catheter (CVC). (A) For gamma-retrovirus, enzyme replacement therapy (ERT) is usually discontinued 14–21 days before gene therapy and patients are treated for 2 days with busulfan. (B) For Lentivirus based HSC-GT, patients are treated for 1 day with busulfan, and ERT is continued during gene therapy until 30 days after infusion.