![]() ![]() ![]() ![]() A good donor match is not always available, though, and there are significant risks, including graft-versus-host disease, which can be very serious. The most common treatment for SCID is a haematopoietic stem cell transplant ( bone marrow transplant) from a matched donor, which allows the child to develop a normal immune system. In the 1970’s, the condition was dubbed ‘bubble boy syndrome’ – named for the experience of SCID patient David Vetter who lived inside a sterile chamber for much of his life. Once diagnosed, children with SCID need to be kept away from all potential sources of infection. These infections will also be less likely to respond to standard treatments and will take longer to resolve. Within six months, there is a high chance of infection because they are unable to make functional lymphocytes (a type of white blood cells essential to immune system function). It is important to understand the precise genetic cause in each case, however, because treatment options vary, and it can have implications for future pregnancies.īabies with SCID usually appear healthy at birth and for a short time afterwards, while they still benefit from their mother’s antibodies. There are a number of such genes, and variants in any of them can lead to very similar symptoms. The condition is caused when a child lacks a functional copy of genes that are essential to creating a working immune system. Without treatment, children with SCID usually die in the first year of life. SCID, is a rare disease in which children are unable to generate immune responses, leaving them vulnerable to infections that would normally be considered harmless. The results of a recent clinical trial, published in The New England Journal of Medicine, showed extremely promising results, with all 50 of the participants surviving after a 24- or 36-month follow-up period. This week, we look at a significant breakthrough for the treatment of severe combined immunodeficiencyĪ new ‘robust’ gene therapy for severe combined immunodeficiency (SCID), a condition which leaves children without an immune system, has been developed by researchers at Great Ormond Street Hospital (GOSH) and the University of California, Los Angeles (UCLA). ![]()
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