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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：
10255294
负责人：
Min Shen
金额：
$30.57万
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/10255294
关键词：
Animal ModelBiological AssayCellsCessation of lifeClassical galactosemiaDevelopmentDietDiseaseEnzymesGalactoseGalactosemiasGalectin 1HumanLeadLeftMeasurementMetabolicMetabolic DiseasesPathogenicityPatientsPharmaceutical ChemistrySeriesSymptomsbasegalactokinaseimprovedinhibitor/antagonistinterestlead seriesliquid chromatography mass spectrometrynovel therapeutics

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中文摘要
翻译
该项目旨在鉴定和开发GALK抑制剂作为经典半乳糖血症的潜在新疗法。在此期间，合作团队继续进行其正在进行的药物化学活动，以（1）改善先导系列的GALK抑制活性，（2）改善备用系列的GALK抑制活性，（2）改善BBB暴露。感兴趣的化合物已被推进到基于细胞的测定和动物模型中，以进一步表征其细胞活性。此外，基于细胞的Gal 1-p LC/MS分析正在开发中，以直接测量细胞中的代谢物，并提供急需的靶向活性标记物。
英文摘要
This project aims to identify and develop inhibitors of GALK as a potential novel therapy for Classic Galactosemia. During this period, the collaborative team continued with its ongoing medicinal chemistry campaign to (1) improve GALK inhibitory activity on the lead series, (2) improve GALK inhibitory activity on the backup series, (2) improve BBB exposure. 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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