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

鉴定可改善自闭症 NLGN4X/Y 运输的小分子

• 批准号: 10688963
• 负责人: Mark Henderson
• 金额: $ 14.62万
• 依托单位: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10688963
• 关键词: Cell Line; Cell surface; Cells; Collaborations; Disease; Extramural Activities; Goals; Informatics; Modeling; Pharmaceutical Chemistry; Process; Proteins; Research; Translations; United States National Institutes of Health; Work; autism spectrum disorder; base; drug discovery; high throughput screening; improved; novel; screening; small molecule; small molecule therapeutics; therapeutic development; trafficking

This collaborative team aims to build on previous research by uncovering novel modulators of NLGN4X/Y trafficking to the cell surface. NLGN4X/Y is a protein that is implicated in autism. The Early Translation Branch's (ETB) overall goal is to uncover new small molecule therapeutics and catalyze the process of therapeutic development through a model of collaborative research. With each collaboration, NIH and extramural disease experts work with the ETBs drug discovery teams, who provide access to small molecule screening, medicinal chemistry and informatics expertise. During this period, the team has developed a strategy for a cell-based high-throughput screen of NLGN4X/Y trafficking and generated cell lines to perform the screen.

该合作团队的目标是在先前研究的基础上，发现NLGN4X/Y转运到细胞表面的新调节剂。NLGN4X/Y是一种与自闭症有关的蛋白质。早期翻译分支（ETB）的总体目标是发现新的小分子治疗方法，并通过合作研究模式催化治疗发展的过程。在每次合作中，NIH和校外疾病专家都与ETBs药物发现小组合作，后者提供小分子筛选、药物化学和信息学专业知识。