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UAB Pilot Center for Precision Animal Modeling (C-PAM) - Disease Modeling Unit

UAB 精密动物建模试点中心 (C-PAM) - 疾病建模单位

基本信息

批准号：
10477314
负责人：
ROBERT A KESTERSON
金额：
$ 90.04万
依托单位：
UNIVERSITY OF ALABAMA AT BIRMINGHAM
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-09-10 至 2025-08-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10477314
关键词：
Alleles Animal Disease Models Animal Model Area Biochemical Genetics Bioinformatics Biological Caenorhabditis elegans Cardiovascular Diseases Cell physiology Clinical Clinical Sciences Communities Data Scientist Development Disease Disease Pathway Disease model Drug Screening Functional disorder Funding Opportunities Generations Genes Genetic Models Genetic Screening Genetic Structures Genetic Variation Genetic study Goals House mice Human Immune System Diseases In Vitro Institute of Medicine (U.S.)Intervention Modeling Molecular Mus Musculoskeletal Diseases Mutagenesis Mutation Neurodevelopmental Disorder Neurologic Pathogenesis Pathogenicity Pathway interactions Patients Phenotype Physiological Play Preclinical Testing Production Rattus norvegicus Research Research Personnel Resources Rodent Rodent Model Role Series Services Standardization Technology Testing Therapeutic Time Translational Research United States National Institutes of Health Variant Vision Zebrafish animal model development base ciliopathy clinical center cost cost effective experience gene function genetic resource genetic variant in vivo in vivo Model innovation insight interest loss of function mutation model development molecular phenotype mutant new technology novel novel therapeutics pre-clinical precision medicine repaired screening therapeutic evaluation treatment center

项目摘要

ABSTRACT (DISEASE MODELING UNIT) The C-PAM Disease Modeling Unit (DMU) supports the UAB Center for Precision Animal Modeling (C-PAM) by creating and validating innovative animal models that recapitulate patient disease at both molecular and phenotypic levels so that we can better understand disease pathogenesis and make highly relevant models ready for preclinical testing. Based on input from Preclinical, Bioinformatics, and Coordinating Sections, the DMU creates patient driven variant alleles in model organisms including C. elegans, zebrafish, mice, and rats to functionally analyze nominated human variants suspected of causing disease. The resulting innovative models are subject to rigorous and standardized phenotypical characterization prior to distribution by the Resource and Services Section. The DMU focuses initially on demonstration projects related to disease models and pathways associated with several areas of strength at UAB including ciliopathies, cohesionopathies, and rasopathies, neurological/ neurodevelopmental, musculoskeletal, cardiovascular, and immunological disorders. Community nominated projects selected for genetic model development begin in year 2 with generated models thoroughly characterized by the team of investigators associated with C-PAM and the nominating clinician or researcher.

摘要(疾病建模单元) C-PAM疾病建模单位(DMU)通过以下方式支持UAB精确动物建模中心(C-PAM) 创建和验证创新的动物模型，从分子和分子两个方面概括患者疾病表型水平，以便我们更好地了解疾病的发病机制，并建立高度相关的模型准备好进行临床前测试。根据临床前、生物信息学和协调部门的投入，DMU 在包括线虫、斑马鱼、小鼠和大鼠在内的模式生物中创建患者驱动的变异等位基因从功能上分析被怀疑导致疾病的指定人类变种。由此产生的创新模式在由资源分发之前，必须经过严格和标准化的表型表征服务组。DMU最初专注于与疾病模型和路径相关的示范项目与UAB的几个优势领域相关，包括纤毛疾病、内聚疾病和类风湿疾病，神经/神经发育、肌肉骨骼、心血管和免疫学疾病。社区被选中用于遗传模型开发的提名项目从第二年开始，彻底地生成模型以与C-PAM相关的研究团队和提名的临床医生或研究人员为特征。