UAB Recessive PKD Research and Translational Core Center

描述：（申请人提供） 位于伯明翰的亚拉巴马大学（UAB）隐性多囊肾病核心中心（DAB RPKDCC）将建立一个基于UAB的跨学科中心，专注于PKD相关研究，特别强调隐性PKD。UAB RPKDCC建立在UAB研究人员的核心骨干基础上，建立了专注于人类ARPKD和/或隐性PKD小鼠模型研究的合作关系。核心中心扩大到包括来自UAB（机构研究基地）的杰出研究人员，以及美国和加拿大的多个机构（扩展研究基地）。集合的研究人员的集体专业知识已被组织成四个主题组：1）纤毛相关的生物学，包括钙介导的机械转导途径; 2）上皮转运途径的调节; 3）上皮分化中至关重要的信号传导途径;和4）基质生物学和纤维化。这些重点领域纳入了隐性PKD发病机制中涉及的主要机制。核心中心的研究人员将受益于获得一套五个互补的生物医学研究核心，将整合大学现有的知识和技术资源，并提供一套服务/资源，使创新的调查在四个主题领域。提出的生物医学核心是：1）核心A -ARPKD临床和遗传资源; 2）核心B -工程小鼠资源; 3）核心C -细胞生理学资源; 4）核心D -组织表征和免疫试剂资源;和5）核心E -蛋白质组学资源。总的来说，这些核心和为该中心聚集的非凡的研究人员队伍将提供对隐性PKD的创新和富有成效的研究至关重要的专业知识的广度和深度。凭借其包括临床和基础研究人员在内的扩展研究基地，该核心中心模式将加速将新的研究见解转化为ARPKD患者的新疗法。

DESCRIPTION: (provided by applicant) The University of Alabama at Birmingham (UAB) Recessive Polycystic Kidney Disease Core Center (DAB RPKDCC) will establish a UAB-based interdisciplinary center of excellence in PKD-related research, with specific emphasis on recessive PKD. The UAB RPKDCC builds on a core cadre of UAB investigators with established collaborations focused on investigations of human ARPKD and/or mouse models of recessive PKD. The Core Center expands to include outstanding investigators from UAB (Institutional Research Base), as well as multiple institutions in the US and Canada (Extended Research Base). The collective expertise of the assembled investigators has been organized into four thematic groups: 1) cilia-related biology, including calcium-mediated mechanotransduction pathways; 2) regulation of epithelial transport pathways; 3) signaling pathways that are critical in epithelial differentiation; and 4) matrix biology and fibrosis. These areas of emphasis incorporate the major mechanisms implicated in the pathogenesis of recessive PKD. The Core Center investigators will benefit from access to a set of five complementary Biomedical Research Cores that will integrate existing intellectual and technological resources of the University and provide a set of services/resources that will enable innovative investigations in the four thematic areas. The proposed biomedical cores are: 1) Core A - The ARPKD Clinical and Genetic Resource; 2) Core B - The Engineered Mouse Resource; 3) Core C - The Cellular Physiology Resource; 4) Core D - The Tissue Characterization and Immunoreagent Resource; and 5) Core E - The Proteomic Resource. Taken together, these Cores and the extraordinary cohort of investigators assembled for this Center will provide the breadth and depth of expertise that is critical for innovative and productive research in recessive PKD. With its Extended Research Base that includes both clinical and basic investigators, this Core Center model will accelerate the translation of new investigative insights toward new therapies for ARPKD patients.