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

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asked Dec 4, 2017 in Biology by mdsamim (213,225 points) 5 10 15
What is gene therapy? Illustrate using the example of adenosine deaminase (ADA) deficiency.

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answered Dec 4, 2017 by sforrest072 (157,439 points) 61 410 947
selected Dec 6, 2017 by sarthaks
 
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Gene therapy is a technique for correcting a defective gene through gene manipulation. It involves the delivery of a normal gene into the individual to replace the defective gene, for example, the introduction of gene for adenosine deaminase (ADA) in ADA deficient individual. The adenosine deaminase enzyme is important for the normal functioning of the immune system. The individual suffering from this disorder can be cured by transplantation of bone marrow cells. The first step involves the extraction of lymphocyte from the patient’s bone marrow. Then, a functional gene for ADA is introduced into lymphocytes with the help of retrovirus. These treated lymphocytes containing ADA gene are then introduced into the patient’s bone marrow. Thus, the gene gets activated producing functional T- lymphocytes and activating the patient’s immune system.

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