Why do children cured by enzyme replacement therapy of adenosine deaminase deficiency, need periodic treatment?[AI 2015]
Answers
Answer:
Cause. Adenosine deaminase deficiency (ADA deficiency) is caused by changes ( mutations ) in the ADA gene . This gene is responsible for making an enzyme that is found in specialized white blood cells ( lymphocytes ). Lymphocytes are an important part of the immune system and help protect the body from infections.
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Answer:
Children cured by enzyme-replacement therapy for adenosine deaminase (ADA) deficiency need periodic treatment because it is not a completely curative method. In this method, a functional ADA, cDNA is injected into a patient, by introducing it into the patient's lymphocytes using a retroviral vector. Since the lymphocytes have a definite life cycle, there is a need for periodic transfusion of the genetically engineered lymphocytes (having ADA) into the patient.