A Novel Small Molecule Targets NKCC1 To Restore Synaptic Inhibition

Trends Pharmacol Sci. 2020 Dec;41(12):897-899. doi: 10.1016/j.tips.2020.10.002. Epub 2020 Oct 20.


Finely tuned excitation-inhibition balance is essential for proper brain function, and loss of balance resulting from reduced synaptic inhibition is associated with neurological disorders. Savardi and colleagues have discovered a novel inhibitor of a cation-chloride transporter that is required for synaptic inhibition, and which restores behaviors associated with Down syndrome (DS) and autism spectrum disorder (ASD).

Keywords: Down syndrome; NKCC1; autism; excitation–inhibition balance; in silico, drug discovery.

Publication types

  • Research Support, Non-U.S. Gov't
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