Send to

Choose Destination
See comment in PubMed Commons below
Front Cell Neurosci. 2013 May 28;7:77. doi: 10.3389/fncel.2013.00077. eCollection 2013.

Developing BACE-1 inhibitors for FXS.

Author information

Department of Neurology, University of Wisconsin Madison, WI, USA.


Fragile X syndrome (FXS) is a debilitating genetic disorder with no cure and few therapeutic options. Excessive signaling through metabotropic glutamate receptor 5 in FXS leads to increased translation of numerous synaptic proteins and exaggerated long-term depression. Two of the overexpressed proteins are amyloid-beta protein precursor (APP) and its metabolite amyloid-beta, which have been well-studied in Alzheimer's disease (AD). Here we discus the possibility that pharmaceuticals under study for the modulation of these proteins in AD might be viable therapeutic strategies for FXS. Specifically, a recently identified acetyltransferase inhibitor that reduces the levels and activity of β-site APP cleaving enzyme (BACE-1) has strong potential to attenuate BACE-1 activity and maintain homeostatic levels APP catabolites in FXS.


Alzheimer’s disease; BACE-1; amyloid-beta; fragile X syndrome; mGluR5

PubMed Commons home

PubMed Commons

How to join PubMed Commons

    Supplemental Content

    Full text links

    Icon for Frontiers Media SA Icon for PubMed Central
    Loading ...
    Support Center