[Enzyme replacement therapy of patients with lysosomal storage disease].
The history and bases of enzyme replacement therapy are briefly reviewed. The enzyme replacement therapy for Gaucher disease type 1, which has been developed for clinical use and is about to be introduced in our country, was described somewhat in detail under the items of the modification of human placental glucocerebrosidase into the macrophage-terminated enzyme, its clinical usage, effects and their evaluations, adverse effects, and new attempts of its application for Gaucher disease types II and III, now being under clinical trials. Also touched are developments of other enzymes for such lysosomal diseases as Fabry disease, Pompe disease, Hurler syndrome, Hunter disease, and Sly disease.
Takada
G
G
Akita University School of Medicine, Department of Pediatrics
Takahashi
T
T
D002240:Carbohydrate Sequence; D005962:Glucosylceramidase / Q000008:administration & dosage* / Q000009:adverse effects / Q000737:chemistry; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000628:therapy*; D008969:Molecular Sequence Data; D010920:Placenta / Q000201:enzymology
D005962:Glucosylceramidase