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X-SCID

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Noun1.X-SCID - SCID in male children resulting from mutation of a gene that codes for a protein on the surface of T cells that allows them to develop a growth factor receptor
SCID, severe combined immunodeficiency, severe combined immunodeficiency disease - a congenital disease affecting T cells that can result from a mutation in any one of several different genes; children with it are susceptible to infectious disease; if untreated it is lethal within the first year or two of life


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Ada-SCID affects both boys and girls and is more difficult to treat than X-SCID.
The Food and Drug Administration (FDA's) Cellular, Tissue and Gene Therapies Advisory Committee made the recommendation following a March 4 hearing called in response to a recent report a third child with X-SCID being treated with gene therapy had developed leukemia in a similar French study.
The agency stressed that both children had been successfully treated by gene therapy for X-SCID and would have died without treatment.
 
 
 
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