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

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
批准号：
10004994
负责人：
Min Shen
金额：
$25.03万
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
项目状态：
未结题
起止时间：
至
来源：
https://reporter.nih.gov/project-details/10004994
关键词：
Animal ModelBiological AssayCellsCessation of lifeClassical galactosemiaDevelopmentDietDiseaseEnzymesGalactoseGalactosemiasGalectin 1HumanLeadLeftMeasurementMetabolicMetabolic DiseasesPathogenicityPatientsPharmaceutical ChemistrySymptomsbasegalactokinaseimprovedinhibitor/antagonistinterestliquid chromatography mass spectrometrynovel therapeutics

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中文摘要
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英文摘要
This project aims to identify and develop inhibitors of GALK as a potential novel therapy for this metabolic disease. During this period, the collaborative team 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. In addition, a cell-based Gal1-p LC/MS assay has been in development to enable direct measurement of the metabolite in cells and provide a much needed marker of on-target activity.
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Identification of Inhibitors of human Galactokinase (GALK) for the treatment of Galactosemia
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