Question

State the possibility of a permanent cure of ADA deficiency disease.

Answer 1

Bone marrow transplantation can cure ADA deficiency in certain children, while enzyme replacement treatment, which involves injecting the patient with functional ADA, can treat it in others. However, the fact that they cannot be totally cured is the issue. A lasting remedy might be possible if the gene extracted from marrow cells that produce ADA is inserted into cells early in the embryonic process.