Question

what is gene therapy? Give example of deficiency of ADA.

Answer 1

Gene therapy is the therapeutic delivery of nucleic acid into a patient's cells as a drug to treat disease. 

Example of deficiency if ADA Lack of normal adenosine deaminase (ADA) activity, a genetic (inherited) condition causing one form of severe combined immunodeficiency ((SCID) disease. It is said to be "combined" in that there is dysfunction of both B and T lymphocytes with impaired cellular immunity and decreased production of immunoglobulins

Answer 2

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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