(a) Mention the cause of ADA deficiency in humans.

(b) How is gene therapy carried out to treat the patients suffering from this disease?

(c) State the possibility of a permanent cure of this disease.

 

 
 
 
 
 

Answers (1)

a) Due to the deletion of the gene for adenosine deaminase in human.

b) Lymphocytes from the blood of the patient are grown in a culture medium outside the body, a functional ADA cDNA (using a retroviral vector) is then introduced into these lymphocytes which are subsequently returned to the patient, the patient requires a periodic infusion of such genetically engineered lymphocytes.

c) If the gene isolated from bone marrow cells producing ADA is introduced into cells at the early embryonic stages then it could be a permanent cure.

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