Abstract:Chronic granulomatous disease (CGD) is a congenital immunodeficiency characterized by lack of reactive oxygen species in phagocytes. We developed an in vivo gene therapy strategy based on intravenous (iv) injection of lentiviral vectors (LVs) in X-CGD mice. A non-myeloablative chemo-conditioning regimen using busulfan, cyclophosphamide and dexamethasone was developed to improve iv LV gene delivery efficiency. The X-CGD mice received two LVs injections. After the second injection, antibody response to LV partic… Show more
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