title: Experimental enzyme replacement in genetic and other disorders.
abstract: To correct a genetic defect, it would not be enough, say, to inject the missing enzyme, since the body's immune defenses would rapidly destroy it. A "Trojan horse" is needed to evade immune surveillance. The evolution of such an approach is described, as well as how it has been used to "cure" Tay-Sachs disease in culture, utilizing immunoglobulin-disguised liposomes to bring hexosaminidase A to deficient cells.
mesh_terms: D000818:Animals; D002478:Cells, Cultured; D003593:Cytoplasm / Q000201:enzymology; D004195:Disease Models, Animal; D057487:Enzyme Therapy; D005818:Genetic Engineering; D006596:Hexosaminidases / Q000172:deficiency; D006801:Humans; D007136:Immunoglobulins; D007962:Leukocytes / Q000201:enzymology; D008064:Lipidoses / Q000628:therapy; D008081:Liposomes; D008247:Lysosomes / Q000201:enzymology; D008661:Metabolism, Inborn Errors / Q000628:therapy*; D009113:Muramidase; D010544:Peroxidases / Q000172:deficiency; D010586:Phagocytes / Q000276:immunology; D012754:Sharks
chemical_list: D007136:Immunoglobulins; D008081:Liposomes; D010544:Peroxidases; D006596:Hexosaminidases; D009113:Muramidase