UAB Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC) - In Vivo Bioassay and Model Development Resource

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ABSTRACT (CORE C) The primary mission of the UAB-Childhood Cystic Kidney Disease Core Center (UAB-CCKDCC) is to work with the PKD Consortium, under the direction of the U24 Central Coordinating Site (U24-CCS) and NIDDK, to eliminate obstacles in cystic kidney disease research that are slowing progress toward the development of improved treatment strategies. The UAB-CCKDCC has assembled a multidisciplinary team of researchers that direct four tightly integrated resource and service-oriented Cores along with an Administrative Core. The collective mission of these Cores is to support, accelerate, and expand basic and translational research by PKD Consortium members by providing access to clinical data and biomaterial from CCKD patients, through the development and distribution of patient-relevant cell and animal models of CCKD to analyze pathogenic mechanisms involved in cyst initiation and progression, and through the development of streamlined, cost- effective pipelines for the PKD Consortium to rapidly ascertain the efficacy of new drugs to slow cyst growth. The In Vivo Bioassay and Model Development Resource (Core C) within the UAB-CCKDCC is tasked with generating and distributing animal models, in vivo biosensors and reporter systems for CCKD associated pathways, and biological reagents generated from these models. Models to be developed include biosensors and reporters in multiple organisms needed to study signaling pathways involved in CCKD and animal models for a wide range of CCKD syndromes with human patient mutations that will be generated based on data obtained in Core A in the UAB-CCKDCC and from the overall PKD Consortium. Core C will also maintain a bank of biomaterials from these models for distribution to promote pilot studies to rapidly test new hypotheses. Finally, the wide spectrum of CCKD and reporter systems generated and maintained by Core C will be made readily available to PKD Consortium for preclinical trails being conducted in Core D of the UAB-CCKDCC to evaluate candidate therapies using highly standardized, cost-effective, and longitudinal imaging and innovative analysis strategies. The services and resources being made available by Core C and it’s integration with the other Cores in the UAB-CCKDCC will expand research activities beyond what is capable in most individual labs and will accelerate research into possible cures by providing for high quality, robust, and reproducible outcomes that are critically needed to prioritize drugs for future clinical trials to halt PKD and other cystic kidney disorders.