Enzyme replacement with liposomes containing beta-galactosidase from Charonia lumpas in murine globoid cell leukodystrophy (twitcher).
Enzyme replacement with liposomes containing beta-galactosidase obtained from charonia lumpas was carried out in murine globoid cell leukodystrophy (GLD). Charonia lumpas beta-galactosidase was able to hydrolyze galactocerebroside trapped into liposomes prepared from lecithin, cholesterol and sulfatide (molar ratio; 7:2:1). Liposomes containing charonia lumpas beta-galactosidase were successfully incorporated into the mouse tissues. 3H-galactocerebroside labeled liposomes were also incorporated into mouse liver, spleen and other tissues. The accumulation rate of 3H-galactocerebroside into twithcer mice liver and spleen was almost 40 to 100 times higher than those of controls and degraded to 70 to 80% of accumulated radioactivity of 3H-galactocerebroside by single injection of liposomes containing charonia lumpas beta-galactosidase. Results suggest that exogeneous enzyme trapped in liposomes can be useful for the correction of accumulated compound.
D000818:Animals; D001236:Aspergillus oryzae / Q000201:enzymology; D001921:Brain / Q000201:enzymology; D002417:Cattle; D002554:Cerebrosides / Q000378:metabolism*; D004195:Disease Models, Animal; D007887:Fabaceae / Q000201:enzymology; D005696:Galactosidases / Q000008:administration & dosage*; D005699:Galactosylceramides / Q000378:metabolism*; D006868:Hydrolysis; D007668:Kidney / Q000201:enzymology; D007965:Leukodystrophy, Globoid Cell / Q000188:drug therapy* / Q000201:enzymology; D008081:Liposomes / Q000008:administration & dosage; D008099:Liver / Q000201:enzymology; D051379:Mice; D008818:Mice, Neurologic Mutants; D008974:Mollusca / Q000201:enzymology; D010946:Plants, Medicinal; D013154:Spleen / Q000201:enzymology; D001616:beta-Galactosidase / Q000008:administration & dosage* / Q000627:therapeutic use
D002554:Cerebrosides; D005699:Galactosylceramides; D008081:Liposomes; C002454:galactocerebroside; D005696:Galactosidases; D001616:beta-Galactosidase