Enzyme-replacement therapy: problems and prospects.

Abstract

Several diseases can, at least in theory, be treated by the administration of an enzyme, the deficiency of which is the cause of the disease. Various attempts have been made to correct enzymatic deficiencies responsible for the clinical manifestation of diseases for which prevention cannot be achieved by modification of the diet or by supportive therapy with drugs. Except for treating certain digestive disorders, enzyme-replacement therapy has not yet found a broad application. In this review article a compilation is given of the problems and prospects of enzyme-replacement therapy in diseases caused by the deficiency of an enzyme.