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Question
Answer the following question.
Two children, A and B aged 4 and 5 years respectively visited a hospital with a similar genetic disorder. The girl A was provided enzyme-replacement therapy and was advised to revisit periodically for further treatment. The girl, B was, however, given a therapy that did not require revisit for further treatment.
Why did the treatment provided to girl A required repeated visits?
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Solution
Girl A was given Enzyme replacement therapy in which lymphocytes isolated from patient's blood are cultured in-vitro. Functional ADA cDNA is then introduced into the cultured lymphocytes. These lymphocytes are returned back to the patient's body. Lymphocytes are not immortal. Therefore, repeated infusion of genetically engineered lymphocytes is required and hence it is not a permanent treatment and the patient has to revisit periodically in the future.
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