Question

Read the following paragraph and answer the questions that follow:

Biotechnology revolves around the "gene of interest", with an objective to open various avenues for human welfare in health, medicine, pharma, agriculture etc. using different techniques, tools and processes. One of the breakthroughs of biotechnology in medicine is the gene therapy.

1. Name the human disease for which the gene therapy was used for the first time.
2. Explain the steps of gene therapy carried to cure the disease using the lymphocytes of the patient. Why is this therapy not a permanent cure of the disease?
3. Write the possible permanent cure of the disease by the gene therapy that is in progress. 

Answer 1

1. The first clinical gene therapy was administered to a 4-year-old girl with ADA deficiency in 1990.
2. The loss of the gene encoding adenosine deaminase, an enzyme essential for the immune system's proper operation, is what causes the illness.
   Treatment:
   • By transferring bone marrow cells, the person who has this condition can be treated.
   • The patient's bone marrow is used to obtain lymphocytes in the first step. Then, with the aid of a retrovirus, a functional gene for ADA is delivered into lymphocytes.
   • The patient's bone marrow is subsequently given these treated cells that have the ADA gene. As a result, the patient's immune system is stimulated and functional T cells are produced by the gene.
   • However, because these cells are not immortal, the patient needs to get these genetically modified lymphocytes on a regular basis.
3. The defect might be permanently cured if a functional ADA gene is extracted from bone marrow cells and inserted at early embryonic stages.