Allotransplantation for the Treatment of Inborn Errors of Metabolism

Excerpt

Possibilities for enzyme replacement therapy in patients with inborn errors of metabolism have drawn increasing interest during the past few years (1)*. The stimulus for this approach has come from the rapid advances in elucidation of the metabolic defects in these disorders, the early and accurate prenatal and postnatal detection of affected persons, the identification of carriers, and the relative ineffectiveness of other treatments in modifying the natural history of many of these diseases.

Two methods for enzyme replacement have been used: direct enzyme infusion and allotransplantation. Enzyme infusions have been tried in a number of inborn errors of metabolism,