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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?
Expand the Given and mention one application of each:
PCR
Recombination DNA−technology is of great importance in the field of medicine. With the help of a flow chart, show how this technology has been used in preparing genetically engineered human insulins.
Expand the Given and mention one application of each:
ELISA
Answer the following question.
Explain the various steps involved in the production of artificial insulin.
Describe the role of high temperature.
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 ____________.
Which one of the following statements is true regarding DNA polymerase used in PCR?
Mention the number of primers required in each cycle of PCR. Write the role of primers and DNA polymerase in PCR. Name the source organism of the DNA polymerase used in PCR.
How is the amplification of a gene sample of interest carried out using PCR?
What is genetically engineered Insulin?
Explain how “Rosie” is different from a normal cow.
ELISA technique is based on the principles of antigen-antibody interaction. Can this technique be used in the molecular diagnosis of a genetic disorder, such as phenylketonuria?
Explain how ADA deficiency can be corrected?
A probe which is a molecule used to locale-specific sequences in a mixture of DNA or RNA Mole cubs could be ______.
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.
How is a mature, functional insulin hormone different from its prohormone form?
When a foreign DNA is introduced into an organism, how is it maintained in the host and how is it transferred to the progeny of the organism?
A patient is suffering from ADA deficiency. Can he be cured? How?
List the advantages of recombinant insulin.
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.
In humans, somatic gene therapy was carried out to correct an immunodeficiency disease. Name this disease.
How many cycles of PCR are required to produce 250 molecules of DNA, starting with a single parental strand?
Atherosclerosis can be treated with recombinant protein called ______
Choose the INCORRECT statement.
