Severe combined immunodeficiency disease, adenosine deaminase deficiency and gene therapy.
Patients with severe combined immunodeficiency disease represent a model for the first clinical applications of gene therapy. Present attempts use insertion of the human adenosine deaminase gene into the peripheral blood T lymphocytes of patients who lack this gene. The ultimate treatment, however, will require insertion of the normal human adenosine deaminase gene into pluripotent stem cells and expression of the gene in their progeny.
Parkman
R
R
Childrens Hospital, Los Angeles, California.
Gelfand
E W
EW
D000243:Adenosine Deaminase / Q000172:deficiency*; D000818:Animals; D004195:Disease Models, Animal; D015316:Genetic Therapy; D051379:Mice; D016511:Severe Combined Immunodeficiency / Q000276:immunology / Q000628:therapy*; D014162:Transfection
D000243:Adenosine Deaminase