Abstract: | In principle, enzyme-loaded erythrocytes can be used as a vehicle for enzyme replacement therapy in lysosomal storage diseases. Glutaraldehyde treatment renders these erythrocytes more resistant to lysis without inactivating the enzymes that have been entrapped inside them. Glutaraldehyde treatment does not prevent ingestion of enzyme-loaded erythrocytes by macrophages in vitro so that these cells can be used to deliver enzymes to lysosomes. In vivo, the glutaraldehyde-treated cells are quickly removed from the circulation by the spleen or liver. The degree of glutaraldehyde treatment allows the erythrocytes to be targeted either to the spleen (low glutaraldehyde concentrations) or to the liver (higher glutaraldehyde concentrations). |