Identification of small molecules that decrease the activity of the huntingtin gene promoter for the potential treatment of Huntington Disease

鉴定降低亨廷顿基因启动子活性的小分子，用于潜在治疗亨廷顿病

• 批准号: 10006272
• 负责人: Juan Marugan
• 金额: $ 13.27万
• 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/10006272
• 关键词: Biology; Chemicals; Collaborations; Communities; Development; Disease; Extramural Activities; Fostering; General Population; Genes; Genomics; Goals; Huntington Disease; Huntington gene; Informatics; Modeling; Pharmaceutical Chemistry; Pharmaceutical Preparations; Phenotype; Process; Publications; Research; Research Personnel; Resources; Synthesis Chemistry; United States National Institutes of Health; Validation; Work; assay development; base; drug development; drug discovery; high throughput screening; human disease; improved; inhibitor/antagonist; lead optimization; new technology; novel; novel therapeutics; promoter; screening; small molecule; small molecule therapeutics; therapeutic development; tool

During this period, the collaborative team worked to characterize and validate hit compounds previously identified in the primary huntingtin screen. As a center, the NCGC has fostered and maintained over 130 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，协作团队致力于表征和验证先前在初级亨廷顿筛选中识别的命中化合物。 作为一个中心，NCGC 与 NIH 和校外研究人员建立并维持了 130 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。