Treatment of Gaucher's disease with liposome-entrapped glucocerebroside: beta-glucosidase.
An adult with Gaucher's disease was injected with liposomes containing human glucocerebroside: beta-glucosidase. After 13 months of treatment the lower border of the right hepatic lobe and moved up 3 cm, and the left lobe had shrunk. Reduction in the size of the liver was associated with relief of pressure symptoms in the left lower abdomen. Scans obtained after the injection of radiolabelled liposomes suggested improvement in function of the reticuloendothelial system.
D000328:Adult; D005260:Female; D005500:Follow-Up Studies; D005776:Gaucher Disease / Q000188:drug therapy*; D005959:Glucosidases / Q000008:administration & dosage*; D005962:Glucosylceramidase / Q000008:administration & dosage* / Q000494:pharmacology; D006801:Humans; D007204:Indium; D007275:Injections, Intravenous; D007553:Isotope Labeling; D008081:Liposomes / Q000008:administration & dosage; D008099:Liver / Q000187:drug effects; D009929:Organ Size / Q000187:drug effects; D014677:Pharmaceutical Vehicles; D010920:Placenta / Q000201:enzymology; D011247:Pregnancy; D011877:Radionuclide Imaging
D008081:Liposomes; D014677:Pharmaceutical Vehicles; D007204:Indium; D005959:Glucosidases; D005962:Glucosylceramidase