Question

Which of the following statements is incorrect about gene therapy in ADA deficiency?

• A. Lymphocytes from patient's blood are taken out and cultured.
• B. A functional ADA cDNA is introduced into these lymphocytes.
• C. Lymphocytes are then introduced in the body of patient.
• D. Patient does not require periodic infusion of genetically engineered lymphocytes.

Answer 1

The correct option is D Patient does not require periodic infusion of genetically engineered lymphocytes.

As a first step towards gene therapy, lymphocytes from the blood of the patient are grown in a culture outside the body. A functional ADA-cNDA (using a retroviral vector) is then introduced into these lymphocytes, which are subsequently returned to the body of the patient. However, as these cells are not immortal, the patient requires periodic infusion of such genetically engineered lymphocytes.

So, the correct answer is 'Patient does not require periodic infusion of genetically engineered lymphocytes'.