SCAVENGER RECEPTOR UPTAKE FOR FABRY DISEASE ENZYME REPLACEMENT THERAPY

摘要

The present invention relates to a composition comprising a lysosomal enzyme conjugated to a negatively charged scavenger receptor ligand. In some embodiments, the lysosomal enzyme is conjugated to the scavenger receptor ligand by way of a linker. The present invention also relates to a composition comprising lysosomal enzyme encapsulated by a liposome, said liposome externally comprising a negatively charged scavenger receptor ligand. The invention further encompasses a method of treating a lysosomal storage disease with the compositions listed above. The invention further encompasses a method of treating a lysosomal storage disease with an acylated, acetylated, or aconitylated lysosomal enzyme.