Q. 48 A patient is suffering from ADA deficiency. Can he be cured? How?

ADA enzyme is crucial for the immune system to function. Deletion of the gene for adenosine deaminase results into ADA deficiency.

In some children, ADA deficiency can be cured by bone-marrow transplantation, while 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 is that they are not completely curative.

It may recurrent in nature as in the process of gene therapy, lymphocytes used are found not to be immortal in nature and the patient requires a periodic infusion of such genetically engineered lymphocytes.

For permanent cure, gene isolated from the bone-marrow cells producing ADA is introduced into cells at early embryonic stages.