Drug – bio-affecting and body treating compositions – Enzyme or coenzyme containing – Stabilized enzymes or enzymes complexed with nonenzyme
Patent
1994-01-14
1995-07-18
Knode, Marian C.
Drug, bio-affecting and body treating compositions
Enzyme or coenzyme containing
Stabilized enzymes or enzymes complexed with nonenzyme
424 946, 424 9461, 435195, 435200, A61K 3847
Patent
active
054339464
ABSTRACT:
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.
REFERENCES:
patent: 4749570 (1988-06-01), Poznansky
Allen et al., (Oct. 15, 1990), "Metabolic Correction of Fucosidosis Lymphoid Cells by Galaptin-.alpha.-L-Fucosidase Conjugates", Biochemical and Biophysical Resource Communications, (172), pp. 335-340.
Poznansky et al., (1989), "Enzyme Replacement Therapy in Fibroblasts from a patient with Cholesteryl Ester Storage Disease", FASEB (3), pp. 152-156.
Ahmed et al., (1990), "Human Splenic Galaptin: Carbohydrate-Binding Specificity and Characterization of the Combining Site", Biochemistry, 29, pp. 5315-5319.
Barton et al., (1990), "Therapeutic Response to intravenous infusions of glycocerebrosides in a patient with Gaucher disease", Proc. Natl. Acad. Sci., 87, pp. 1913-1916.
Hasholt et al., 1988, Clinical Genetics, 33, pp. 360-371.
Fukuda, M., (1985), Biochimica et Biophysica Acta, 780, pp. 119-150.
Allen, Jr. Howard J.
DiCioccio Richard A.
Health Research , Inc.
Knode Marian C.
Witz Jean C.
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