title: Metabolic correction of fucosidosis lymphoid cells by galaptin-alpha-L-fucosidase conjugates.
abstract: To determine if isolated galaptin, an endogenous galactoside-binding lectin, could serve as a transport vehicle of therapeutic agents to cells, galaptin and alpha-L-fucosidase were coupled using glutaraldehyde. The conjugates were incubated with alpha-L-fucosidase-deficient, EBV-immortalized lymphoid cells from a fucosidosis patient. Conjugates were effectively bound and internalized by the cells in a lactose inhibitable manner. Internalization of conjugate resulted in the reduced accumulation of alpha-L-fucosyl-N-acetylglucosaminylasparagine, a glycopeptide that accumulates in cells of fucosidosis patients, to levels found in lymphoid cells from a healthy individual. Thus, galaptin-alpha-L-fucosidase conjugates may be useful for enzyme replacement therapy of fucosidosis. The concept of using galaptin as a transport vehicle may be applied to the delivery of other compounds to cells bearing galaptin receptors.
authors.affiliation: Department of Surgical Oncology, Roswell Park Cancer Institute, Buffalo, NY 14263.
authors.forename: H J
authors.initials: HJ
authors.lastname: Allen
authors.forename: H
authors.initials: H
authors.lastname: Ahmed
authors.forename: R A
authors.initials: RA
authors.lastname: DiCioccio
mesh_terms: D001402:B-Lymphocytes / Q000187:drug effects / Q000378:metabolism; D002352:Carrier Proteins / Q000378:metabolism; D002460:Cell Line; D005645:Fucosidosis / Q000378:metabolism*; D037161:Galectins; D006388:Hemagglutinins / Q000378:metabolism* / Q000494:pharmacology; D006801:Humans; D007700:Kinetics; D012016:Reference Values; D005644:alpha-L-Fucosidase / Q000378:metabolism* / Q000494:pharmacology
chemical_list: D002352:Carrier Proteins; D037161:Galectins; D006388:Hemagglutinins; D005644:alpha-L-Fucosidase