title: Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency.
abstract: 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.
authors: Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Department of Health and Human Services, Bethesda, MD 20892-1456, USA.
authors: Javier
authors: J
authors: Chinen
authors: Joie
authors: J
authors: Davis
authors: Suk See
authors: SS
authors: De Ravin
authors: Beverly N
authors: BN
authors: Hay
authors: Amy P
authors: AP
authors: Hsu
authors: Gilda F
authors: GF
authors: Linton
authors: Nora
authors: N
authors: Naumann
authors: Effie Y H
authors: EY
authors: Nomicos
authors: Christopher
authors: C
authors: Silvin
authors: Jean
authors: J
authors: Ulrick
authors: Narda L
authors: NL
authors: Whiting-Theobald
authors: Harry L
authors: HL
authors: Malech
authors: Jennifer M
authors: JM
authors: Puck
mesh_terms: D015316:Genetic Therapy / Q000379:methods*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D007109:Immunity / Q000187:drug effects*; D007223:Infant; D009154:Mutation; D015375:Receptors, Interleukin-2 / Q000008:administration & dosage* / Q000235:genetics; D012190:Retroviridae / Q000235:genetics; D013601:T-Lymphocytes / Q000276:immunology; D014161:Transduction, Genetic; D014182:Transplantation, Autologous; D053632:X-Linked Combined Immunodeficiency Diseases / Q000235:genetics / Q000276:immunology* / Q000628:therapy*
chemical_list: D015375:Receptors, Interleukin-2