Question

A child in your colony is suffering from ADA deficiency. The doctor has advised two methods to correct this genetic defect. Gene therapy is an attempt to correct this genetic defect by providing a normal gene into the individual. By this the normal function can be restored. Alternatively, enzyme replacement therapy can also restore the function. Which in your opinion is a better option?

Answer 1

In my opinion, gene therapy is a better option to correct ADA deficiency because it aims at a permanent cure by introducing a normal gene into the patient's cells, enabling the body to produce the required enzyme continuously. This corrects the root cause of the disease. The genetically engineered lymphocytes can produce the enzyme and restore immune function, although periodic infusion may be needed if cells are not immortal. In contrast, enzyme replacement therapy provides only temporary relief, as it requires frequent injections and does not cure the disorder. Thus, gene therapy has a more lasting potential benefit.