The genetic defect, adenosine deaminase (ADA) deficiency may be cured permanently by
(a) administering adenosine deaminase through injection
(b) bone marrow transplantation
(c) enzyme replacement therapy
(d) introducing isolated gene from marrow cells producing ADA into the cells at early embryonic stages
Answer (d) : Adenosine deaminase enzyme is crucial for the immune system to function. The disorder ADA deficiency is caused due to deletion of the gene for adenosine deaminase.
ADA deficiency can be cured by bone marrow transplantation or by enzyme replacement therapy, in which functional ADA is given to the patient by injection. But both of these approaches are not completely curative as the patient requires periodic infusion of such genetically engineered lymphocytes or hormonal injections. However, if the gene isolated from marrow cells producing ADA is introduced into cells at early embryonic stages, it could be a permanent cure.
