Clinical and experimental transplantation in enzymatic deficiency disease.

Allotransplantation provides another strategy for the treatment of metabolic diseases. In disorders which primarily affect a specific tissue or organ, appropriate allografts have already proved therapeutically effective. Most of these diseases presumably result from dominantly inherited structural genetic defects. Metabolic transplantation, the grafting of tissues for the continuous production of the normal gene products, can provide effective treatment in appropriate, inherited, metabolic diseases. However, it must be emphasized that this approach is exploratory, and further studies in experimental model systems must be continued. Future advances in immunology and transplantation biology may increase the potential effectiveness of allotransplantation for the treatment of metabolic diseases.