Identification of Inhibitors of human Galactokinase (GALK) for the treatment of Galactosemia

用于治疗半乳糖血症的人半乳糖激酶 (GALK) 抑制剂的鉴定

基本信息

批准号：
9551913
负责人：
Min Shen
金额：
$ 25.16万
依托单位：
NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES
依托单位国家：
美国
项目类别：
财政年份：
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/9551913
关键词：
Animal Model Biological Assay Biology Cells Cessation of life Chemicals Classical galactosemia Collaborations Communities Development Diet Disease Enzymes Extramural Activities Fostering Galactose Galactosemias General Population Genomics Goals Gut associated lymphoid tissue Human Informatics Lead Left Metabolic Metabolic Diseases Modeling Pathogenicity Patients Pharmaceutical Chemistry Pharmaceutical Preparations Phenotype Process Publications Research Research Personnel Resources Symptoms Synthesis Chemistry United States National Institutes of Health Validation Work assay development base drug development drug discovery galactokinase high throughput screening human disease improved inhibitor/antagonist interest new technology novel therapeutics screening small molecule small molecule therapeutics therapeutic development tool

项目摘要

During this period, the NCGC continued with its ongoing medicinal chemistry campaign to improve GALK inhibitory activity. Compounds of interest have been advanced into cell-based assays and animal models to further characterize their cellular activity. 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继续进行了正在进行的药物化学运动，以改善GALK抑制活性。感兴趣的化合物已进入基于细胞的测定和动物模型中，以进一步表征其细胞活性。作为一个中心，NCGC与NIH和壁外研究人员培养并维持了110多次积极合作，从而促进了整个人类疾病范围内的药物发现工作。这些努力导致了数十个高通量屏幕和许多药物化学运动，以进一步改善筛选效果，为我们的合作者和一般研究社区提供出版物以及各种有希望的小分子探测器和铅。此外，NCGC努力促进许多信息计划，以更好地利用现有的药物和疾病目标信息，并为公众提供易于获得的资源，从而进一步促进针对人类疾病的新疗法的发展。