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

Answers (1)

Gene therapy refers to a collection of methods and technologies allowing correction or replacement of a defective or dysfunctional gene. While correcting, a normal gene is transferred into the individual's embryo to replace and perform the function of the original defective gene. One well-established example of gene therapy is ADA deficiency. This is caused by the deletion of a gene encoding for enzyme adenosine deaminase.  The gene therapy for ADA deficiency was carried out in a 4-year-old patient by the following method:

1. The lymphocytes of a patient were grown in a culture in vitro.

2. A functional ADA cDNA was introduced in these lymphocytes using a retroviral vector.

3. The transformed lymphocytes were introduced back into the blood of the patient.

4. The patient is regularly infused with transformed lymphocytes 

5. For a permanent cure of ADA deficiency, a gene isolated from bone marrow cell is introduced in the patient in the early stage. 

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