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

NCATS早期翻译分支（ETB）拥有一个广泛而全面的计划，用于发现针对罕见疾病的候选药物和探索人类基因组功能的药理学工具。ETB开展研究，以了解推动基础研究发现转化为人类健康切实改善的基本原则。 在本报告所述期间，合作团队完成了对约50，000种化合物的高通量筛选，以鉴定能够改善NLGN 4X/Y向细胞表面运输的小分子，并鉴定了几种感兴趣的化学型。 还进行了分子建模和虚拟筛选研究，通过这项工作鉴定的化合物正在调查中。

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.