Expression of cDNA-encoded human acid a-glucosidase in milk of transgenic mice

摘要

Enzyme replacement therapy is at present the option of choice lor treatment of lysosomal storage diseases. To explore the feasibility of lysosomal enzyme production in milk of iransgonic animals, the human acid a-glucosidase cDNA was placed under control of the us)"i:asciii promoter and expressed in mice. The milk contained reeomhinnnt enzyme at a concentration up to 1.5 u,g/ml. Enzyme purified from milk of transgenie mice was internalized via the mannose 6-phosphate receptor and corrected enzyme deficiency in I'ihmblasts from patients. We conclude that transgenically produced human acid a-glucosidase meets the criteria for therapeutic application.