Question 5: What is gene therapy? Illustrate using the example of adenosine deaminase (ADA) deficiency.
Class 12 - Biology - Biotechnology and its Applications Page 216
Answers
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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Answer:
gene therapy is a medical field which focuses on the utilization of the therapeutic delivery of nucleic acid into a patient's cells as a drug to treat disease.
eg.of ADA deficiency are:chronic diarrhea,widespread skin rashes ,and etc