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A child is born with ADA-deficiency.(a) Suggest and explain a procedure for possible life-long (permanent) cure. (b) Name any other possible treatment for this disease. |
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Answer» (a) Gene isolated from marrow cells producing ADA is introduced into cells at early embryonic stages, it could be a permanent cure. (b) ADA deficiency can be cured by bone marrow transplantation or by enzyme replacement therapy, in which functional ADA is given to the patient by injection. Another option is gene therapy in which lymphocytes from the blood of the patient are grown in a culture outside the body. A functional ADA cDNA (using a retroviral vector) is then introduced into these lymphocytes, which are subsequently returned to the patient . |
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