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Prospects for gene therapy for immunodeficiency diseases.

The fields of gene transfer and expression have recently seen major advances that have made possible a better understanding of basic biologic mechanisms and have brought closer the possibility for gene therapy for the treatment of clinical disease. Vectors derived from murine retroviruses are being developed for use in clinical protocols. In this article we address the recent advances in gene transfer as they relate to progress toward achieving gene therapy for immunodeficiency diseases, specifically for adenosine deaminase (ADA) deficiency as a cause of severe combined immunodeficiency (SCID). It now appears that ADA deficiency will be the first disease in which the technique of retroviral-mediated gene therapy will be attempted.