Abstract
A recent report by Gaspar et al. summarizes the clinical long-term follow-up (LTFU) of 10 patients presenting with X-linked severe combined immunodeficiency (SCID-X1) who were treated with first-generation gamma-retroviral vectors to reconstitute the expression of the interleukin-2 receptor common γ-chain (IL2RG) in autologous hematopoietic cells.1 As in a related study of nine patients conducted by Hacein-Bey-Abina et al.,2 the children enrolled in this study had no human leukocyte antigen–matched donor, thus reducing the safety and effectiveness of an allogeneic stem cell transplant.3,4 In both studies, gene-modified hematopoietic cells were infused without prior myelosuppressive conditioning. Despite some differences in the design of vector backbones, envelope proteins, and transduction conditions, these studies share five important major findings.1,2
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