Retroviral-mediated transfer and expression of the common gamma chain intohuman hematopoietic progenitors

The common gamma chain (gamma(c)) of cytokine receptors is mutated in X-lin ked severe combined immunodeficiency, a lethal disorder characterized by th e absence of both humoral and cellular immune defenses, Allogeneic bone mar row transplantation from HLA-identical siblings usually results in complete reconstitution of the immune system and is the current treatment of choice . Genetic correction and reinfusion of autologous hematopoietic stem cells represents an alternative therapeutic approach for those patients who lack suitable marrow donors. In this study, we show that retroviral-mediated tra nsfer of the gamma(c) gene results in efficient expression in CD34+ cells a nd high transduction rate of colony-forming progenitors.