Two children, A and B aged 4 and 5 years respectively visited a hospital with a similar genetic disorder. The girl A was provided enzyme-replacement therapy and was advised to revisit periodically for further treatment. The girl, B was, however, given atherapy that did not require revisit for further treatment.
(a) Name the ailments the two girls were suffering from?
(b) Why did the treatment provided to girl A required repeated visits?
(c) How was the girl B cured permanently?
(a) The disease mentioned here is ADA (adenosine deaminase) deficiency which is caused due to mutation in gene that code for ADA enzymes. The deficiency of these enzymes causes destruction of B and T lymphocytes which results in weak immune system.
(b) Girl A must be given Enzyme Replacement Therapy in which lymphocytes are isolated from patient’s body and then cultured in-vitro. In the cultured lymphocytes, the ADA cDNA are introduced and then inserted back into patient’s body. But since lymphocytes die after a certain period of time, repeated infusions are given to the patient.
(c) The Girl B must be given the Gene Therapy in which the gene isolated from bone marrow cells producing ADA is introduced into cells at embryonic stages which is a permanent cure.
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