1-Deoxy-D-galactonojirimycins with dansyl capped N-substituents as Î²-galactosidase inhibitors and potential probes for GM1 gangliosidosis affected cell lines.

[gm1 gangliosidosis]

Two simple and reliably accessible intermediates , N-carboxypentyl- and N-aminohexyl-1 -deoxy-D-galactonojirimycin were employed for the synthesis of a set of terminally N-dansyl substituted derivatives . Reaction of the terminal carboxylic acid of N-carboxypentyl-1 -deoxy-D-galactonojirimycin with N-dansyl-1 ,6 -diaminohexane provided the chain-extended fluorescent derivative . Employing bis (6 -dansylaminohexyl )amine , the corresponding branched di -N-dansyl compound was obtained . Partially protected N-aminohexyl-1 -deoxy-D-galactonojirimycin served as intermediate for two additional chain-extended fluorescent 1 -deoxy-D-galactonojirimycin (1 -DGJ ) derivatives featuring terminal dansyl groups in the N-alkyl substituent . These new compounds are strong inhibitors of d-galactosidases and may serve as leads en route to pharmacological chaperones for GM 1 -gangliosidosis .

Diseases
Validation

Diseases presenting "additional chain-extended fluorescent" symptom

gm1 gangliosidosis

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