title: Lymphocyte gene therapy.
abstract: Genetically corrected T cells are currently under investigation as a treatment for severe combined immunodeficiency disease resulting from a lack of adenosine deaminase (ADA). Monthly injections of these ADA-corrected T cells have resulted in measurable ADA activity in the peripheral blood and the in vivo production of antibody to blood group antigen. Genetically corrected T cells appear to be clinically valuable vehicles for gene therapy.
authors.affiliation: National Institutes of Health, Bethesda, MD 20892.
authors.forename: K W
authors.initials: KW
authors.lastname: Culver
authors.forename: W F
authors.initials: WF
authors.lastname: Anderson
authors.forename: R M
authors.initials: RM
authors.lastname: Blaese
mesh_terms: D000243:Adenosine Deaminase / Q000172:deficiency / Q000235:genetics*; D002478:Cells, Cultured; D002648:Child; D002985:Clinical Protocols; D015316:Genetic Therapy; D006801:Humans; D016511:Severe Combined Immunodeficiency / Q000201:enzymology / Q000235:genetics / Q000628:therapy*; D013601:T-Lymphocytes / Q000201:enzymology* / Q000637:transplantation; D014162:Transfection
chemical_list: D000243:Adenosine Deaminase