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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
How did an American Company, Eli Lilly use the knowledge of r-DNA technology to produce human insulin?
Compare and contrast the advantages and disadvantages of the production of genetically modified crops.
State the cause of ADA deficiency in humans.Mention a possible cure for an ADA deficiency patient.
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.
Describe the role of high temperature.
The first clinical gene therapy was done for the treatment of ________.
How many amino acids are arranged in the two chains of Insulin?
PCR proceeds in three distinct steps governed by temperature, they are in order of ____________.
PCR is a useful tool for early diagnosis of an Infectious disease. Elaborate.
Human insulin is being commercially produced forma transgenic species of ______.
The site of production of ADA the body is ______.
A pathophysiology is the ______.
Due to which of the following reasons insulin extracted from pancreas of slaughtered animals cannot be used in humans to treat diabetes?
When gene targetting involving gene amplification is attempted in an individual’s tissue to treat disease, it is known as ______
Now a days it is possible to detect the mutated gene causing cancer by allowing a radioactive probe to hybridize its complimentary DNA in a clone of cells, followed by its detection using autoradiography because ______
What is a recombinant DNA vaccine? Give two examples.
Who was the first patient who was given gene therapy? Why was the given treatment recurrent in nature?
Define Antigen and Antibody. Name any two diagnostic kits based upon them.
How is a mature, functional insulin hormone different from its prohormone form?
A patient is suffering from ADA deficiency. Can he be cured? How?
Name the five key tools for accomplishing the tasks of recombinant DNA technology. Also mention the functions of each tool.
A cell-free method of amplifying DNA first developed in the mid 1980's revolutionised the field of biotechnology, Name the method and explain the basic steps of the technique involved.
Explain how recombinant human insulin was prepared in 1983 by Eli Lily an American company.
The graph given below indicates the administration of the first (L) and second dose (M) of a vaccine. The corresponding response of the body is indicated by X, and Y. Interpret the graph and explain the reason for such a response shown by the body.

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?
