Synthesis and utilization of therapeutic agents for the treatment of lysosomal storage diseases
作者： Richard A. DiCioccio , Howard J. Allen
DOI:
关键词: Enzyme replacement therapy 、 Enzyme assay 、 Enzyme 、 Biology 、 Mutant 、 Genetic enhancement 、 Biochemistry 、 Endogeny 、 Antisense therapy
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 treatment lysosomal storage diseases. In accordance with one embodiment, lectin-glycosidase conjugates provided enzyme replacement therapy cells containing deficient activity. another comprising lectin-nucleic acid constructs gene A third embodiment relates to lectin-oligonucleotide antisense modulate production mutant defective enzymes.
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