Identification of small molecules that increase activity of the huntingtin antisense promoter for the potential treatment of Huntington Disease

鉴定可增加亨廷顿蛋白反义启动子活性的小分子，用于潜在治疗亨廷顿病

• 批准号: 9205663
• 负责人: Marc Ferrer
• 金额: $ 1.9万
• 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/9205663
• 关键词: Identification; small; molecules; increase; activity

During this period, the NCGC has fostered and maintained over 180 active collaborations with both NIH and extramural investigators, facilitating drug discovery efforts across the entire spectrum of human disease. These efforts have led to over 100 high-throughput screens and nearly 60 medicinal chemistry campaigns, providing our collaborators and the general research community a wealth of publications and promising small molecule leads. In addition, the NCGC has undertaken a number of informatic challenges 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 与 NIH 和校外研究人员建立并维持了 180 多项积极合作，促进了整个人类疾病范围的药物发现工作。 这些努力已经促成了 100 多个高通量筛选和近 60 个药物化学活动，为我们的合作者和一般研究界提供了丰富的出版物和有前途的小分子先导化合物。 此外，NCGC还承担了多项信息挑战，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。