Question

Adenosine deaminase (ADA) deficiency is a hereditary disease, where ADA, which is crucial for functioning of immune system is absent. Explain how ADA deficiency can be treated.

Answer 1

Lymphocytes are taken from patient’s blood. It is grown invitro culture. By using retroviral vector functional ADA c DNA (from WBC of normal person) is introduced into invitro cultured lymphocytes of patients. These genetically engineered lymphocytes (corrected functional lymphocytes) are re injected to patients. If the ADA producing genes are introduce into cells at early embryonic stage it could be a permanent cure.