A drug-screening platform for autism spectrum disorders using human neurons and astrocytes

使用人类神经元和星形胶质细胞的自闭症谱系障碍药物筛选平台

• 批准号: 9360486
• 负责人: Marc Ferrer
• 金额: $ 3.75万
• 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/9360486
• 关键词: Astrocytes; Behavioral; Biological Assay; Biology; Chemicals; Collaborations; Communities; Development; Disease; Extramural Activities; Fostering; General Population; Genomics; Goals; Human; Impairment; Informatics; Lead; Methyl-CpG-Binding Protein 2; Modeling; Mutation; Neurons; Occupational; Patients; Pharmaceutical Chemistry; Pharmaceutical Preparations; Preclinical Drug Evaluation; Process; Publications; Research; Research Personnel; Resources; Rett Syndrome; Series; Synthesis Chemistry; United States National Institutes of Health; Validation; Work; assay development; autism spectrum disorder; base; drug development; drug discovery; high throughput screening; human disease; improved; new technology; novel therapeutics; screening; small molecule; small molecule therapeutics; social; therapeutic development; tool

During this period, the NCGC has worked to adapt and optimize an HTS-amenable human neuron assay, and high-throughput screening is underway. As a center, the NCGC has fostered and maintained over 110 active collaborations with both NIH and extramural investigators, facilitating drug discovery efforts across the entire spectrum of human disease. These efforts have led to dozens of high-throughput screens and a number of medicinal chemistry campaigns to further improve on screening hits, providing our collaborators and the general research community with publications and a variety of promising small molecule probes and leads. In addition, the NCGC has worked to advance a number of informatic initiatives to make better use of existing drug and disease target information and provide the general public with easily accessible resources, further catalyzing the development of new therapies for human disease.

在此期间，NCGC 一直致力于调整和优化适用于 HTS 的人类神经元检测，并且高通量筛选正在进行中。 作为一个中心，NCGC 与 NIH 和校外研究人员建立并维持了 110 多项积极合作，促进了整个人类疾病范围的药物发现工作。 这些努力导致了数十次高通量筛选和许多药物化学活动，以进一步提高筛选命中率，为我们的合作者和一般研究界提供出版物和各种有前途的小分子探针和先导化合物。 此外，NCGC还致力于推进多项信息化举措，以更好地利用现有药物和疾病靶点信息，为公众提供易于获取的资源，进一步促进人类疾病新疗法的开发。