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High-throughput screening to identify modulators of PKD2 function

High-throughput screening to identify modulators of PKD2 function
高通量筛选鉴定 PKD2 功能调节剂
批准号：
10255323
负责人：
Mark Henderson
金额：
$11.24万
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/10255323
关键词：
Autosomal Dominant Polycystic KidneyBiological AssayBiologyCationsCell membraneCellsCollaborationsComplexDiseaseExtramural ActivitiesGoalsInformaticsIntegral Membrane ProteinModelingMutationPharmaceutical ChemistryPhenotypeProcessProprotein Convertase 1ResearchResearch PersonnelSynthesis ChemistryTranslationsUnited States National Institutes of HealthValidationWorkassay developmentbasedesigndisease-causing mutationdrug developmentdrug discoveryhigh throughput screeninglead optimizationnew technologynovelpolycystic kidney disease 1 proteinscreeningsmall moleculesmall molecule therapeuticstherapeutic developmenttool

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中文摘要
翻译
常染色体显性多囊肾病（ADPKD）是由跨膜蛋白PKD 1（PC 1）或PKD 2（PC 2）突变引起的。 PC 1和PC 2亚基在质膜上形成阳离子通道，其功能可被致病突变破坏 在此期间，该团队致力于设计和实施一种高通量筛选检测方法，能够识别PC 1/PC 2异聚体复合物的小分子激活剂。 Delling实验室开发了一种新的基于细胞的PC 1/PC 2活性测定法。
英文摘要
Autosomal dominant polycystic kidney disease (ADPKD) is caused by mutations in transmembrane proteins PKD1 (PC1) or PKD2 (PC2). PC1 and PC2 subunits form a cation channel at the plasma membrane, and function can be disrupted by disease-causing mutations During this period, the team worked to design and implement a high-throughput screening amenable assay capable of identifying small molecule activators of the PC1/PC2 heteromeric complex. A novel cell-based assay of PC1/PC2 activity was developed by the Delling lab.
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