Personalized Model System Core

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PROJECT SUMMARY: A key component of this P30 application is the ‘Personalized Model System Core’. The models will include the patient-specific cultures derived from intestinal epithelia (enteroids) and the induced pluripotent stem cells (iPSCs) from myofibroblasts. These patient specific cultures will be used to study CFTR function and fluid homeostasis, and the information obtained will be used towards personalized treatment options. The Personalized Model System core will develop and bank these models, develop and validate the assays for CF/CFTR research, and distribute the resources to researchers worldwide. The long-term goal of this core is to use models derived from the patient to facilitate his/her personalized therapy. Specific Aim 1. Will be to provide CF patient-specific enteroids (small bowel and colonic). Specific Aim 2. Will be to provide well-characterized CF patient-specific induced pluripotent stem cells (iPSCs)-derived tissue from myofibroblasts. Specific Aim 3. Will provide assay services and training to local, national and international investigators interested in personalized model system to study the functional involvement of CFTR fluid secretion in intestinal enteroids, spheroids and organoids. The Core will create resources and sound services that have important long term clinical implications toward patient-specific therapies for cystic fibrosis. The core will provide training in a variety of state-of-art and innovative techniques including the development of intestinal stem cells cultures, iPSCs technology, high content microscopy, to name a few, to researchers who will be able to use these techniques to study not only CF/CFTR biology but other membrane proteins and their associated diseases.