Question

Gene therapy can be used to treat an ADA deficient patient. Explain.
Is it a permanent cure? How can ADA be cured permanently?

Hint:

ADA deficiency can be treated temporarily by gene therapy or permanently through early-stage gene insertion.

Answer 1

Gene therapy involves the insertion of functional ADA gene into the lymphocytes of the patient. These cells are grown in vitro and returned to the patient’s body. This restores the ADA enzyme temporarily.
Permanent cure: It can be achieved by introducing the functional gene into cells at the embryonic stage, ensuring a lifelong supply of ADA.