Q22 of 43 Page 1

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.


a) Gene therapy is the procedure through which a permanent solution can be reached. In this procedure lymphocytes from the blood of a patient are grown in a culture outside the body, functional ADA cDNA is introduced into these lymphocytes, these cells are returned to the patient’s body at early embryonic stage.


b) Other possible treatments for ADA-deficiency are: bone marrow transplant (for which an exact match would have to be found) and enzyme replacement therapy.


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