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प्रश्न
A patient is suffering from ADA deficiency. Can he be cured? How?
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उत्तर
The first clinical gene therapy was given in 1990 to a 4-year-old girl with adenosine deaminase (ADA) deficiency. This enzyme is crucial for the immune system to function. The disorder is caused due to the deletion of the gene for adenosine deaminase. In some children ADA deficiency can be cured by bone marrow transplantation; in others it can be treated by enzyme replacement therapy, in which functional ADA is given to the patient by injection. But the problem with both of these approaches that they are not completely curative. As a first step towards gene therapy, lymphocytes from the blood of the patient are grown in a culture outside the body. A functional ADA cDNA (using a retroviral vector) is then introduced into these lymphocytes, which are subsequently returned to the patient. However, as these cells are not immortal, the patient requires periodic infusion of such genetically engineered lymphocytes. However, if the gene isolate from marrow cells producing ADA is introduced into cells at early embryonic stages, it could be a permanent cure.
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संबंधित प्रश्न
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.
Explain how insulin can be produced using recombinant DNA technology.
Give a schematic representation of the transformation of a pro-insulin into insulin.
The first clinical gene therapy was done for the treatment of ________.
A patient suffering from diabetes mellitus will have ______.
A probe which is a molecule used to locale-specific sequences in a mixture of DNA or RNA Mole cubs could be ______.
ADA is an enzyme which is deficient in a genetic disorder SCID. What is the full form of ADA?
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.
Give the full form of ELISA. Which disease can be detected using it? Discuss the principle underlying the test.
What is a recombinant DNA vaccine? Give two examples.
Define Antigen and Antibody. Name any two diagnostic kits based upon them.
Explain how recombinant human insulin was prepared in 1983 by Eli Lily an American company.
- Assertion: PCR is a powerful technique to identify genetic disorders.
- Reason: PCR can detect mutations in low amounts of DNA.
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?
The ______ gene from Bacillus thuringiensis produces a protein that forms crystalline inclusions in bacterial spores.
Atherosclerosis can be treated with recombinant protein called ______
Globally, approximately how many recombinant therapeutics have been approved for human use, and how many of them are currently marketed in India?
In the recombinant production of humulin using Escherichia coli, which plasmid vector and bacterial gene are fused with the human insulin A and B chain sequences?
