Ontology highlight
ABSTRACT:
SUBMITTER: Dawson G
PROVIDER: S-EPMC2881699 | biostudies-literature | 2010 Apr
REPOSITORIES: biostudies-literature
Dawson Glyn G Schroeder Christina C Dawson Philip E PE
Biochemical and biophysical research communications 20100325 1
Competitive inhibitors of lysosomal hydrolases (pharmacological chaperones) have been used to treat some lysosomal storage diseases which result from mis-sense mutations and mis-folded protein but have not been tried in Batten disease, for which there is no current therapy. We synthesized a large number of novel, non-hydrolyzable competitive inhibitors of palmitoyl:protein thioesterase (PPT1) and showed that some could act as chemical chaperones. One inhibitor (CS38: betaAGDap(Pal)VKIKK) was tak ...[more]