Enzyme Replacement with Liposomes Containing β-Galactosidase in Murine Krabbe's Globoid Cell Leukodystrophy

Abstract

Enzyme replacement with liposomes containing β-galactosidase obtained from charonia lumpas was carried out in murine globoid cell leukodystrophy (OLD) . Charonia lumpas β-galactosidase was able to hydrolyze galactocerebroside and trapped into liposomes prepared from lecithin, cholesterol and sulfatide (molar ratio; 7: 2: 1) . Liposomes containing charonia lumpas β-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 Twitcher 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-galactocere-broside by single injection of liposomes containing charonia lumpas β-galactosidase. Results suggest that exogeneous enzyme trapped in liposomes can be useful for the correction of accumulated compound.