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Question
Consider the following statements (A-D) and select the option stating which ones are true (T) and false (F).
A. SCID patients have functional T- lymphocytes.
B. The patients cannot mount immune responses against invading pathogens.
C. Ideal approach for SCID treatment would be to give the patient genetically engineered lymphocytes with functional ADA genes.
D. The first clinical gene therapy was given in 1995.
Consider the following statements (A-D) and select the option stating which ones are true (T) and false (F).
A. SCID patients have functional T- lymphocytes.
B. The patients cannot mount immune responses against invading pathogens.
C. Ideal approach for SCID treatment would be to give the patient genetically engineered lymphocytes with functional ADA genes.
D. The first clinical gene therapy was given in 1995.
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Solution
The correct option is A A - F, B - T, C - T, D - F
The mutation of the gene coding for the enzyme adenosine deaminase (ADA) can cause SCID (Severe Combined Immunodeficiency). ADA is involved in purine metabolism. When DNA is broken down, it generates a molecule known as deoxyadenosine which is toxic to lymphocytes. The enzyme ADA is crucial for the immune system to function as it is involved in the conversion of deoxyadenosine to a non-toxic molecule called deoxyinosine. The patients with ADA deficiency, as a result, would have non-functional lymphocytes and so they cannot mount immune responses against invading pathogens.
The ideal approach for SCID treatment would be to give patients a functioning ADA that breaks down toxic biological products. Lymphocytes from the blood of the patient are grown in a culture outside the body. A good copy of human gene encoding a functional ADA cDNA is introduced into the lymphocytes (collected from patients) by using retroviral vectors. The modified lymphocytes are subsequently returned to the patient.
The first clinical gene therapy was given by French Anderson and Michael Blaese in 1990 to a 4 year old girl suffering from SCID due to Adenosine deaminase (ADA) deficiency.
The mutation of the gene coding for the enzyme adenosine deaminase (ADA) can cause SCID (Severe Combined Immunodeficiency). ADA is involved in purine metabolism. When DNA is broken down, it generates a molecule known as deoxyadenosine which is toxic to lymphocytes. The enzyme ADA is crucial for the immune system to function as it is involved in the conversion of deoxyadenosine to a non-toxic molecule called deoxyinosine. The patients with ADA deficiency, as a result, would have non-functional lymphocytes and so they cannot mount immune responses against invading pathogens.
The ideal approach for SCID treatment would be to give patients a functioning ADA that breaks down toxic biological products. Lymphocytes from the blood of the patient are grown in a culture outside the body. A good copy of human gene encoding a functional ADA cDNA is introduced into the lymphocytes (collected from patients) by using retroviral vectors. The modified lymphocytes are subsequently returned to the patient.
The first clinical gene therapy was given by French Anderson and Michael Blaese in 1990 to a 4 year old girl suffering from SCID due to Adenosine deaminase (ADA) deficiency.
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