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प्रश्न
What is gene therapy? Illustrate using the example of adenosine deaminase (ADA) deficiency.
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उत्तर
Gene therapy is a genetic engineering approach that involves replacing a faulty gene with a normal, functional gene. In 1990, a 4-year-old child suffering from adenosine deaminase deficiency received the first clinical gene therapy. This enzyme is essential for the immune system to operate. Severe combined immunodeficiency (SCID) is caused by a gene abnormality in the enzyme adenosine deaminase. SCID patients lack functioning T-lymphocytes and cannot fight bacteria and other microorganisms.
Treatment of ADA deficiency using gene therapy.
To perform gene therapy, doctors take lymphocytes from the patient’s bone marrow and use a retroviral vector to introduce a normal, functional copy of the human ADA gene into those lymphocytes. The treated cells are reintroduced into the patient’s bone marrow. These cells produce lymphocytes that carry the functional ADA gene, which reactivates the victim’s immune system. However, due to their limited lifespan and inability to divide, these lymphocytes require regular replenishment with engineered cells. Modifying stem cells during the early embryonic stage can solve this difficulty.
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संबंधित प्रश्न
Why do children cured by enzyme-replacement therapy adenosine deaminase deficiency need periodic treatment?
Suggest any two possible treatments that can be given to a patient exhibiting adenosine deaminase deficiency.
State the role of C-peptide in human insulin.
Diagrammatically represent the experimental steps in cloning and expressing an human gene (say the gene for growth hormone) into a bacterium like E. coli?
Explain how insulin can be produced using recombinant DNA technology.
State the best-known contribution of P.K. Sethi
Give a schematic representation of the transformation of a pro-insulin into insulin.
Write any two biochemical/molecular diagonostic procedures for early detection of viral infection. Explain the principle of any one of them.
Answer the following question.
Describe the roles of heat, primers, and the bacterium Thermus aquaticus in the process of PCR.
The first clinical gene therapy was done for the treatment of ________.
The genetic defect adenosine deaminase deficiency may be cured permanently by ____________.
How is the amplification of a gene sample of interest carried out using PCR?
What is genetically engineered Insulin?
Explain how ADA deficiency can be corrected?
What are DNA vaccines?
Explain how recombinant Insulin can be produced.
A pathophysiology is the ______.
ADA is an enzyme which is deficient in a genetic disorder SCID. What is the full form of ADA?
Choose the correct option regarding retrovirus.
When gene targetting involving gene amplification is attempted in an individual’s tissue to treat disease, it is known as ______
With regard to insulin choose the correct options.
- C-peptide is not present in mature insulin.
- The insulin produced by rDNA technology has C-peptide.
- The pro-insulin has C-peptide.
- A-peptide and B-peptide of insulin are interconnected by disulphide bridges.
Choose the correct answer from the options given below.
For effective treatment of the disease, early diagnosis and understanding of its pathophysiology is very important. Which of the following molecular diagnostic techniques is very useful for early detection?
Give the full form of ELISA. Which disease can be detected using it? Discuss the principle underlying the test.
Can a disease be detected before its symptoms appear? Explain the principle involved.
What is a recombinant DNA vaccine? Give two examples.
In humans, somatic gene therapy was carried out to correct an immunodeficiency disease. Name this disease.
The diagram given below represents the schematic structure of proinsulin, which undergoes certain modifications before it becomes a fully functional insulin.
Study the diagram carefully and answer the questions that follow:
- State the change the proinsulin undergoes to become fully functional.
- Name the modern scientific technique used for the production of human insulin.
- How are the two polypeptide chains of the fully functional insulin held together?
How many cycles of PCR are required to produce 250 molecules of DNA, starting with a single parental strand?
Name the technique used to detect the presence of HIV in the body of an individual, Justify the principle associated with this technique.
