✦ LIBER ✦

Severe combined immunodeficiency disease, adenosine deaminase deficiency and gene therapy

✍ Scribed by Robertson Parkman; Erwin W Gelfand

Publisher: Elsevier Science
Year: 1991
Tongue: English
Weight: 741 KB
Volume: 3
Category: Article
ISSN: 0952-7915
DOI: 10.1016/0952-7915(91)90019-w

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✦ Synopsis

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.

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