TREATMENT OF ADENOSINE-DEAMINASE DEFICIENCY

Severe combined immune deficiency disease due to a deficiency of the e nzyme adenosine deaminase is a rare disease. However, it has been used as a prototype disease for the development of a variety of treatment modalities that are currently applied in more frequent diseases. For e xample, allogeneic bone marrow transplantation and stem cell gene ther apy have been used for adenosine deaminase deficiency before being app lied in other more frequent diseases. In the present paper, the develo pment of bone marrow transplantation and stem cell gene therapy, as we ll as treatment with purified enzyme, for adenosine deaminase deficien cy are discussed.