ADA-SCID


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Related to ADA-SCID: Adenosine deaminase deficiency
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Noun1.ADA-SCID - SCID resulting from mutation of a gene that codes for adenosine deaminaseADA-SCID - SCID resulting from mutation of a gene that codes for adenosine deaminase
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
References in periodicals archive ?
In ADA-SCID, a single genetic defect prevents children from developing a vigorous immune system, leaving them unable to fight off ordinary infections.
Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement.
The clinical course of inherited ADA-SCID ranges from the rapidly fatal to the minimally dysfunctional immune system of patients presenting 'partial' ADA deficiency (7).
It recommended less stringent criteria to allow resumption of the single gene therapy study aimed at a related immune system disorder called ADA-SCID, and also that the agency allow 27 distantly related gene therapy experiments to proceed under strict informed consent guidelines and closely followed for signs of leukemia.
Gene therapy has seen success in the treatment of diseases such as myelination disorder X-linked ALD (adenoleukodystrophy), ADA-SCID (adenosine deaminase deficiency-related severe combined immunodeficiency), and Leber congenital amaurosis in discrete trials.