Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency
Department of Pediatrics
Medical Subject Headings
Gene Therapy; Hematopoietic Stem Cell Transplantation; Humans; Immunity; Infant; Mutation; Receptors, Interleukin-2; Retroviridae; T-Lymphocytes; Transduction, Genetic; Transplantation, Autologous; X-Linked Combined Immunodeficiency Diseases
Genetics and Genomics | Medical Genetics | Pediatrics
Retroviral gene therapy can restore immunity to infants with X-linked severe combined immunodeficiency (XSCID) caused by mutations in the IL2RG gene encoding the common gamma chain (gammac) of receptors for interleukins 2 (IL-2), -4, -7, -9, -15, and -21. We investigated the safety and efficacy of gene therapy as salvage treatment for older XSCID children with inadequate immune reconstitution despite prior bone marrow transplant from a parent. Subjects received retrovirus-transduced autologous peripherally mobilized CD34(+) hematopoietic cells. T-cell function significantly improved in the youngest subject (age 10 years), and multilineage retroviral marking occurred in all 3 children.
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Citation: Blood. 2007 Jul 1;110(1):67-73. Epub 2007 Mar 16. Link to article on publisher's site