METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF beta-GLUCOCEREBROSIDASE.

摘要

Provided is a method of increasing the stability of wild-type beta- glucocerebrosidase. Also provided are methods of treating and/or preventing an individual having a neurological disease in which increased expression or activity of beta-glucocerebrosidase in the central nervous system would be beneficial. This method comprises administering an effective amount of a pharmacologic chaperone for beta- glucocerebrosidase, with the proviso that the individual does not have a mutation in the gene encoding beta-glucocerebrosidase. Further provided are beta-glucocerebrosidase inhibitors which have been identified as specific pharmacologic chaperones and which have been shown to increase activity of beta-glucocerebrosidase in vivo in the central nervous system.