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adenosine deaminase deficiency
(redirected from ADA deficiency)

   Also found in: Wikipedia 0.01 sec.
adenosine deaminase deficiency
ADA deficiency A uniformly fatal AD disease, which consitutes 40% of Pts with SCID Clinical Cellular immune dysfunction, oral candidiasis, intractable diarrhea, FTT, severe diaper rash, pseudoachondrodysplasia, death by age 2 Lab ↓ Lymphocytes < 0.5 x 109/L–US, < 500 mm3, especially T cells; eosinophilia; ↑ adenosine and deoxyadenosine in serum and urine Treatment Gene therapy; rarely BMT. See Cartilage-hair syndrome.


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Children whose SCID is due to ADA deficiency have another alternative.
1990: The first gene therapy takes place, on a four-year-old girl with an immune-system disorder called ADA deficiency.
Most children with ADA deficiency can be treated with bone marrow transplants or regular injections of the missing enzyme.
 
 
 
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