What is gene therapy? Illustrate using the example of adenosine deaminase (ADA) deficiency.
Gene Therapy. It is defined as the introduction of a normal functional gene into cells which contain the defective gene with the objective of correcting a gene defect that has been diagnosed in a child/embryo.
For example - Treatment of ADA deficiency by gene therapy. Adenosine deaminase is an enzyme crucial for funtioning of immune system. Gene therapy has been used to cure the deficiency of ADA enzyme
1. By growing the lymphocyte from blood cells of patient in culture outside the body.
2. A funtional ADA gene is introduced in the cultured lymphocyte using retrovirus.
3.The transformed lymphocytes are then returned to the patient.
4. Thus the cells now contain funtional gene for the enzyme and deficiency is cured.
