Discovery of small molecule activators of the Slack potassium channel for use as cell-based probes for the study of fragile X syndrome

Project Details


PROJECT NARRATIVE Fragile X syndrome (FXS) is a genetic disorder that is the most common inherited cause of intellectual disability and often comorbid with autism spectrum disorder (ASD). Epigenetic silencing of the FMR1 gene in FXS results in a deficiency of the Fragile X mental retardation protein (FMRP), a binding protein that activates Slack potassium channels. Drugs that activate Slack channels may provide effective treatments for FXS, and potent and selective Slack activator cell-based probes are required to validate this therapeutic approach.
Effective start/end date1/12/2031/10/22


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