Lymphocyte gene therapy.
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.
Culver
K W
KW
National Institutes of Health, Bethesda, MD 20892.
Anderson
W F
WF
Blaese
R M
RM
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
D000243:Adenosine Deaminase