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Institute of Child Health, London
Gene therapy for severe combined immunodeficiency shows promise
January 9th, 2005
A U.K. study provides further evidence that gene therapy can be effective in creating a functional immune system for infants with severe combined immunodeficiency (SCID). SCID is a genetic disease that prevents the immune system - particularly the white blood cells - from functioning. This leaves individuals at high risk of infection, as documented many years ago by the patient, David Vetter, who lived in a "bubble" for 12 years to prevent infection from the external environment. In the latest study, published in the Lancet (364(9452)), four children with SCID had stem cells removed from their own bone marrow; the relevant "good" gene was...
Source: Aging & Elder Health Week (2005-01-09)
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