| abstract |
A compound represented by General Formula I: ** Formula ** or a pharmaceutically acceptable salt thereof, wherein: each of R1, R2, and R3 is independently a monosaccharide, halide, hydroxyl, amine, or oligosaccharide residue, X is oxygen or sulfur; R4 is (S) -4-amino-2-hydroxybutyryl (AHB); R5 is hydroxyl; Y is hydrogen or alkyl; and the dashed line indicates an R configuration or an S configuration, and wherein at least one of R1, R2 and R3 is a monosaccharide residue represented by General Formula II: ** Formula ** in which: the dashed line indicates an R configuration or an S configuration; and R6, R7, and R8 are each independently selected from the group consisting of hydroxyl and amine, for use in treating a genetic disorder. |