Potent chemical chaperone compounds for GM1-gangliosidosis: N-substituted (+)-conduramine F-4 derivatives
2015
The development of second-generation valienamine-type chemical chaperones for GM1-gangliosidosis is described. Several N-substituted (+)-conduramine F-4 derivatives were designed as novel chemical chaperones based on a lead chaperone compound, N-octyl-4-epi-β-valienamine, to decrease the inhibitory activity while increasing the enzyme enhancement activity. Among the derivatives synthesized during this study, a conduramine derivative with an N-cyclohexylmethyl group has demonstrated significant enhancement of R201C mutated β-galactosidase activity.
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