Identify curative method (s) used to treat ADA deficiency disease in human.
A. Gene Therapy
B. Bone Marrow Transplantation
C. Complete Blood Replacement
D. Enzyme Replacement Therapy
Choose the correct answer from the options given below:
Answer & explanation
Correct answer: option 1
The correct answer is Option (1) → A, B and D only
A. Gene Therapy — True. Lymphocytes given a functional ADA cDNA are put back into the patient.
B. Bone Marrow Transplantation — True. One of the treatments for ADA deficiency.
C. Complete Blood Replacement — False. This is not a treatment for ADA deficiency.
D. Enzyme Replacement Therapy — True. Functional ADA is given by injection.
NCERT Extract:
Gene Therapy – “… 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.”