Preclinical Models Core (Core A)

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PROJECT SUMMARY – PRECLINICAL MODELS CORE The Preclinical Models Core (PMC) will provide access to state-of-the art tools and services, education and training programs and develop new methodologies focused on the discovery and early validation of therapeutic targets for kidney diseases. Despite recent breakthroughs that have led to major advances in our understanding of the pathogenesis of kidney diseases such as polycystic kidney disease and focal segmental glomerulosclerosis, new therapies to diagnose, manage and cure these disorders have been slow to emerge. A key step in the path to provide new treatments for kidney patients is the availability of sophisticated preclinical model systems for basic discovery research, interrogation of newly identified pathways and early target validation. In the PMC, we will exploit the extensive experience and technical expertise of our talented kidney investigators while leveraging substantial Northwestern resources and bring them together under one administrative umbrella to establish and provide easy access to: 1) NUmousekidney housing over 50 unique genetic kidney disease mouse strains 2) a genome editing `concierge' service to rapidly design and generate new genetic renal models 3) non-genetic kidney disease models including hypertensive and renal transplantation models and state-of-the art mouse phenotyping services and 4) service for the generation and genomic editing of induced pluripotent stem cells (iPSC) from patients with kidney diseases and an iPSCkidney biobank. The PMC Core will also provide hands-on workshops and web-based training in mouse genetics, genome editing, induced pluripotent stem cell (iPSC) technologies and phenotyping to kidney investigators and trainees. Outreach and access to all Core-related services will be provided to the global kidney community through an online web portal named nephro-HUB. Seamless integration and strong synergy with the Therapeutics Development and Clinical Translation NU-GoKIDNEY Cores, the Pilot and Feasibility and Enrichment Programs and an innovative Kidney Cure Accelerator program will catalyse discoveries and accelerate their translation into innovative therapies for kidney patients.