CAS: 54322-38-2; 4-Methyl-7-(((2S,3S,4R,5S,6S)-3,4,5-Trihydroxy-6-Methyltetrahydro-2H-Pyran-2-yl)Oxy)-2H-Chromen-2-One

上下游产品

2,3,4-tri-O-acetyl-α-L-fucopyranosyl bromide 7-hydroxy-4-methyl-chromen-2-one 1,2,3,4-tetra-O-acetyl-L-rhamnopyranose

专利信息

专利号:US-5433946-A
优先权日:1991-10-11
标 题 :Synthesis and utilization of therapeutic agents for the treatment of lysosomal storage diseases
发明人：ALLEN JR HOWARD J; DICIOCCIO RICHARD A
权利人:HEALTH RESEARCH INC
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.