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

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
依托单位国家:
美国
项目类别:
财政年份:
--
资助国家:
美国
项目状态:
未结题
起止时间:
至

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中文摘要
翻译
在此期间,合作团队致力于表征和验证先前在初级亨廷顿蛋白筛选中鉴定的命中化合物。 作为一个中心,NCGC与NIH和校外研究人员建立并保持了130多个积极合作,促进了整个人类疾病领域的药物发现工作。 这些努力已经导致了数十个高通量筛选和一些药物化学活动,以进一步改善筛选命中,为我们的合作者和一般研究社区提供出版物和各种有前途的小分子探针和线索。 此外,NCGC还致力于推进多项信息化举措,以更好地利用现有的药物和疾病靶点信息,并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。
英文摘要
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.
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