CYSTIC FIBROSIS RELATED DISEASE MODEL CORE - Core 3- Naren

Cincinnati Childrens Hosp Med Ctr, Cincinnati OH

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Abstract

PROJECT SUMMARY: A new component of this P30 application is the ‘CF-Related Disease Model Core”. This core will include patient- and iPSC-derived CF organ model systems integrated with chip technology that form a highly relevant testing platform for investigators interested in specific questions of human CF-related disease phenotype. The Core will be located at Cedars Sinai Medical Center (CSMC) and will work closely with Core 1 (Clinical and Translational core) and Core 2 (Model System Core) at CCHMC and UC to develop a strong East Coast-West Coast Cystic Fibrosis (CF) partnership. Locally, we will also partner with the Division of Regenerative Medicine and GI Division (CSMC) to make our core accessible to all the researchers interested in CF research in Sothern California as part of the CFF-RDP (SoCal CF-Research Program: a partnership of 5 institutions, Cedars, USC, CHLA, UCLA and Ventura). It is our expectation that access to these innovative state-of-the-art technologies will facilitate translational research at CSMC, CCHMC and UC investigators with an emphasis on CF/CFTR related disorders while also addressing demographic-associated health disparities and outcomes. Two specific aims will be studied: Aim 1: The Core will provide well-characterized CF patient-specific primary pancreatic and iPSC-derived organoid cultures to local, national, and international investigators interested in CF-related disease models. Aim 2: The core will provide CF-organ-on-a-chip service (both commercial and customized) to local, national, and international investigators interested in CF-related model systems. The proposed studies are highly significant because it (i) addresses life-limiting and prevalent diseases and pathological manifestations that limit therapeutic outcomes; (ii) has clinical relevance and implications; (iii) is a multidisciplinary project covering basic biomedical engineering, physiology and pathophysiology of the pancreas, development of tissue on a chip, assay development, and drug discovery using personalized human model systems.