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What is gene therapy? Illustrate using the example of adenosine deaminase (ADA) deficiency.

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प्रश्न

What is gene therapy? Illustrate using the example of adenosine deaminase (ADA) deficiency.

विस्तार में उत्तर
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उत्तर

Gene therapy is a genetic engineering approach that involves replacing a faulty gene with a normal, functional gene. In 1990, a 4-year-old child suffering from adenosine deaminase deficiency received the first clinical gene therapy. This enzyme is essential for the immune system to operate. Severe combined immunodeficiency (SCID) is caused by a gene abnormality in the enzyme adenosine deaminase. SCID patients lack functioning T-lymphocytes and cannot fight bacteria and other microorganisms.


Treatment of ADA deficiency using gene therapy.

To perform gene therapy, doctors take lymphocytes from the patient’s bone marrow and use a retroviral vector to introduce a normal, functional copy of the human ADA gene into those lymphocytes. The treated cells are reintroduced into the patient’s bone marrow. These cells produce lymphocytes that carry the functional ADA gene, which reactivates the victim’s immune system. However, due to their limited lifespan and inability to divide, these lymphocytes require regular replenishment with engineered cells. Modifying stem cells during the early embryonic stage can solve this difficulty.

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अध्याय 10: Biotechnology and its Applications - EXERCISES [पृष्ठ १८७]

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एनसीईआरटी Biology [English] Class 12
अध्याय 10 Biotechnology and its Applications
EXERCISES | Q 8. | पृष्ठ १८७
नूतन Biology [English] Class 12 ISC
अध्याय 12 Biotechnology and Its Applications
NCERT EXERCISES WITH ANSWERS | Q 5. | पृष्ठ ५५६

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