Question
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.

Answer

a. Gene therapy can be used for the treatment of the child with ADA deficiency. In this method, the lymphocytes from the blood of the patient are grown in a culture outside the body. A functional ADA CDNA is introduced into these lymphocytes using a retroviral vector. These lymphocytes are then returned to the patient. If the gene isolated from marrow cells producing ADA is introduced into cells at early embryonic stage, then it can be a permanent cure.
b. Other possible methods for curing ADA deficiency disease Bone marrow transplantation and enzyme replacement therapy (giving the functional enzyme to the patient by injection).

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