MODIFYING EXOGENOUS GLUCOCEREBROSIDASE FOR EFFECTIVE REPLACEMENT THERAPY IN GAUCHER DISEASE

Important therapeutic principles were established in developing effect ive enzyme replacement therapy for patients with Gaucher disease. The background and sequence of the investigations that led to effective de livery of exogenous glucocerebrosidase to the lipid-storing macrophage s in patients with Guacher disease are described. The principle of tar geting the intravenously injected enzyme to the mannose lectin on the surface of these cells by engineering the glycoform of the enzyme is a useful model of an essential requirement for effective enzyme therapy . Similar strategies are expected to be effective for the treatment of a number of hereditary metabolic disorders of humans.