Article
Candidate molecules for chemical chaperone therapy of GM1-gangliosidosis.
Future medicinal chemistry - 1 Sept 2013
Higaki Katsumi, Ninomiya Haruaki, Suzuki Yoshiyuki, Nanba Eiji
Abstract excerpt
A growing body of evidence suggests that misfolding of a mutant protein followed by its aggregation or premature degradation in the endoplasmic reticulum is one of the main mechanisms that underlie inherited neurodegenerative diseases, including lysosomal storage diseases. Chemical or pharmacological chaperones are small molecules that bind to and stabilize mutant lysosomal enzyme proteins in the endoplasmic...
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