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

7/8/2015 CBSE



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






Answer :


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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