Question

A person is born with a hereditary disease with a weakened immune system due to deficiency of an enzyme. Suggest a technique for complete cure for this disease, identify the deficient enzyme and explain the technique used for cure.

Answer 1

Gene therapy can completely cure this disease. 

The disease is due to ADA (Adenosine deaminase) deficiency. 

Lymphocytes from the blood of the patient are grown in a culture. A functional ADA cDNA is introduced into these lymphocytes, which are subsequently returned to the patient. The permanent cure is done by introducing ADA cDNA into cells at early embryonic stages.