Published in Gene Therapy Weekly, November 6th, 1995
Severe combined immunodeficiency (SCID) as a result of inherited deficiency of ADA is usually fatal unless the affected children are kept in protective isolation or if their immune system is not reconstituted by bone marrow transplantation. Many patients do not have an HLA-identical sibling donor to make this particular cure possible. Alternative, hopefully curative therapies are desperately needed.
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