Question

Describe the gene therapy produce for an ADA-deficient patient.

Answer 1

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In gene therapy, lymphocytes from the blood of the patient are grown in a culture outside the body. Adenosine deaminase is an enzyme for purine metabolism. It is essential for lymphocyte development, survival, and function. ADA deficiency is treated by introduction of functional ADA (adenosine deaminase) cDNA (using a retroviral vector) into lymphocytes which are subsequently transferred to the patient. This cDNA is transcribed to m-RNA which is further translated into Adenosine deaminase protein.However as these cells are not immortal, patient requires periodic infusions of such genetically engineered lymphocytes.