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Q.

Which of the following could be a permanent cure for ADA deficiency in a patient?
 

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a

Enzyme replacement therapy by intramuscular injection of ADA enzyme

b

Injection of ADA-corrected lymphocytes to the patient.
 

c

Introduction of a functional ADA cDNA into cells at early embryonic stages
 

d

Bone marrow transplantation

answer is D.

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Detailed Solution

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In somatic gene therapy, a functional ADA cDNA (using a retroviral vector) is introduced into the lymphocytes, which are subsequently returned to the patient. As these cells are not immortal, the patient requires periodic infusion of such genetically engineered lymphocytes. However, if the gene isolate from marrow cells producing ADA is introduced into cells at early embryonic stages, it could be a permanent cure. 

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