Which among the following is a permanent cure for ADA( adenosine deaminase ) deficiency ?
Answer & explanation
Correct answer: option 3
The disorder is caused due to the deletion
of the gene for adenosine deaminase. In some children ADA deficiency
can be cured by bone marrow transplantation; 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.. However, if
the gene isolate from marrow cells producing ADA is introduced into cells
at early embryonic stages, it could be a permanent cure.