A patient is suffering from ADA deficiency. Can he be cured? How?
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ADA (i.e., adenosine deaminase) deficiency is a genetic disorder caused by the deletion of a gene for adenosine deaminase synthesis which leads to the deficiency of the ADA enzyme in the body, which causes damage to the immune system. It can be cured by bone marrow transplantation or enzyme replacement therapy. These do not cure the deficiency completely. Another approach is like growing lymphocytes outside the body of the patient in cell culture and using a functional ADA complementary DNA (i.e., cDNA) in those lymphocytes. If this procedure is done at an embryonic stage, then it can be cured permanently.