Identification of small molecules that improve trafficking of NLGN4X/Y for autism

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The NCATS Early Translation Branch (ETB) hosts a broad and comprehensive program for the discovery of drug candidates directed towards rare diseases and pharmacological tools to probe the function of the human genome. ETB conducts research to understand the underlying principles driving the translation of basic research discoveries into tangible improvements in human health. During this reporting period, the collaborative team completed high-throughput screening of about 50,000 compounds to identify small molecules capable of improving trafficking of NLGN4X/Y to the cell surface, and several chemotypes of interest have been identified. Molecular modeling and virtual screening studies have also been performed, and compounds identified through this work are under investigation.