Synthesis of N-substituted ε-hexonolactams as pharmacological chaperones for the treatment of N370S mutant Gaucher disease.

A series of N-substituted ε-hexonolactams have been designed and prepared by a concise route with a tandem ring-expansion reaction as the key step. Some of the N-substituted ε-hexonolactams show better enhancements to N370S mutant β-glucocerebrosidase activity than NB-DNJ and NN-DNJ. Both the experi...

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Autori principali: Wang, G, Twigg, G, Butters, T, Zhang, S, Zhang, L, Ye, X
Natura: Journal article
Lingua:English
Pubblicazione: 2012