Synthesis and utilization of therapeutic agents for the treatment of lysosomal storage diseases

摘要

The present invention provides compositions wherein endogenous lectins are used as transport vehicles for facilitating the cell-specific binding and cellular uptake of therapeutic agents useful in the treatment of lysosomal storage diseases. In accordance with one embodiment, lectin-glycosidase conjugates are provided for enzyme replacement therapy of cells containing deficient enzyme activity. In accordance with another embodiment, compositions comprising lectin-nucleic acid constructs are provided for gene therapy of cells containing deficient enzyme activity. A third embodiment of the present invention relates to lectin-oligonucleotide compositions provided for antisense therapy to modulate production of mutant and defective lysosomal enzymes.