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AssigneeHealth Research, Inc.
InventorsHoward J. Allen, Jr., Richard A. DiCioccio
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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.
A lectin-enzyme conjugate consisting essentially of human galaptin as a lectin targeting agent, and human .alpha.-L-fucosidase as a functional lysosomal enzyme, wherein the galaptin-.alpha.-L-fucosidase conjugate is internalized by human fucosidosis lymphoid cell-associated galaptin receptor-mediated endocytosis;
A method for treating human cells in need of .alpha.-L-fucosidase enzyme replacement therapy, said method comprising contacting said cells with a therapeutically effective amount of a lectin-enzyme conjugate consisting essentially of human galaptin as a lectin targeting agent, and human .alpha.-L-fucosidase as a functional lysosomal enzyme, wherein the galaptin-.alpha.-L-fucosidase conjugate
Dependencies are inferred from the claim text.