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

Description

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.
StatusActive
Effective start/end date1/12/2031/10/22

Funding

  • National Institute of Mental Health: $170,841.00

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