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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.
Give a schematic representation of the transformation of a pro-insulin into insulin.
Describe the role of bacterium Thermus aquaticus in carrying the process of polymerase chain reaction.
The genetic defect adenosine deaminase deficiency may be cured permanently by ____________.
How was Insulin obtained before the advent of rDNA technology? What were the problems encountered?
The hormone insulin is secreted by:
ADA is an enzyme which is deficient in a genetic disorder SCID. What is the full form of ADA?
Which is false about antibiotics?
Which of the following is the correct sequence of steps in a PCR (Polymerase Chain Reaction)?
Which of the following is not an application of PCR (Polymerase Chain Reaction)?
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?
Define Antigen and Antibody. Name any two diagnostic kits based upon them.
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.
How does a gene therapy involving direct modification of the cells, in order to achieve a therapeutic goal is used in the treatment of ADA deficiency? Explain.
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.
Give a reason for the following:
Streptokinase is administered to the patients having myocardial infarction.
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?
