Question
Download Solution PDFIn gene therapy of Adenosine Deaminase (ADA) deficiency, the patient requires periodic infusion of genetically engineered lymphocytes because:
Answer (Detailed Solution Below)
Detailed Solution
Download Solution PDFConcept:
- Adenosine Deaminase (ADA) is an enzyme required for the proper functioning of the immune system.
- ADA deficiency is caused due to deletion of the gene for the enzyme adenosine deaminase.
Explanation:
- ADA deficiency can be treated through gene therapy.
- In gene therapy, the lymphocytes from the patient are grown outside the body and a functional ADA cDNA is introduced (using a retroviral vector) into the lymphocytes.
- The genetically engineered lymphocytes are then introduced back into the patient.
- These genetically engineered lymphocytes are immortal hence, the patient has to be infused with such lymphocytes regularly.
So, the correct answer is option 1.
Additional Information:
- Introducing genes for ADA into the cells at early embryonic development can lead to a permanent cure for ADA deficiency.
Last updated on Jun 16, 2025
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