Title
Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency
UMMS Affiliation
Department of Pediatrics
Publication Date
2007-07-21
Document Type
Article
Subjects
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
Disciplines
Genetics and Genomics | Medical Genetics | Pediatrics
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.
DOI of Published Version
10.1182/blood-2006-11-058933
Source
Blood. 2007 Jul 1;110(1):67-73. Epub 2007 Mar 16. Link to article on publisher's site
Journal/Book/Conference Title
Blood
Related Resources
PubMed ID
17369490
Repository Citation
Chinen J, Davis J, De Ravin S, Hay BN, Hsu AP, Linton GF, Naumann N, Nomicos EY, Silvin C, Ulrick J, Whiting-Theobald NL, Malech HL, Puck JM. (2007). Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency. Genetics. https://doi.org/10.1182/blood-2006-11-058933. Retrieved from https://escholarship.umassmed.edu/peds_genetics/14