Genetic variants in a protein called NBCn2 have been tied to epilepsy and autism spectrum disorder for years. Until three weeks ago, nobody had seen what the protein looked like, and nobody had a compound that could switch it off.
Researchers at the Icahn School of Medicine at Mount Sinai now have both, and their first inhibitors of the protein are already reducing neuronal activity in the dish. Their work, published in Nature Communications on July 22, reports the first high-resolution structure of NBCn2, along with a series of compounds that inhibit it.