In gene therapy of adenosine deaminase (ADA) deficiency, the patient requires a periodic infusion of…

In gene therapy of adenosine deaminase (ADA) deficiency, the patient requires a periodic infusion of genetically engineered lymphocytes because:
  1. Gene isolated from marrow cells producing ADA is introduced into cells at embryonic stages
  2. Lymphocytes from patient's blood are grown in culture, outside the body.
  3. Genetically engineered lymphocytes are not immortal cells.
  4. Retroviral vector is introduced into these lymphocytes.

Solution

In gene therapy of the ADA, we transform the lymphocytes with the functional copy of the ADA. This process is done inside the lab in the cell culture set up.

These cells are then injected back in the patient. However, these cells are not immortal and will die after their life span is over.

Thus, the patient will need periodic infusion of the genetically engineered lymphocytes.

Asked in: NEET 2022 (Phase 1)

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