Question

Why do children cured by enzyme-replacement therapy for adenosine deaminase deficiency need periodic treatment?

Answer 1

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.