IDD Models Core

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The IDD Models Core is designed to foster development and analysis of human stem cell and rodent models of IDD conditions. The core supports basic science studies of molecular pathways and cellular interactions that are altered in genetic and epigenetic disorders affecting nervous system function. In addition, the core enables drug and biomarker studies, as well as preclinical studies that are aimed at developing novel translational approaches to IDD. A historic strength of the IDD Models core is the use of human pluripotent stem cell (hPSC)-based models, which include both patient-specific induced pluripotent stem cells (iPSCs) and their modification by genome editing, to study diverse types of IDD conditions. The core has incorporated novel approaches to meet the increased demands for rigor and reproducibility by meeting international/peer institution standards in the creation and characterization of patient-specific iPSCs and hESCs. The refined gene editing capabilities develop innovative cellular tools for sophisticated analysis of altered nervous system development and function. These models also provide the means to evaluate candidate therapeutics in disorder-relevant cells, as well as develop novel screens to identify promising compounds in small- and large-scale library screening. The core also empowers analysis of rodent models of IDD conditions, not only by providing support for their creation, but also by providing a comprehensive and cutting edge behavioral testing service. The service incorporates a battery of cognitive/nervous system analyses under the supervision of a skilled core manager to ensure the rigor and reproducibility of these IDD model studies. Our specific aims for the next project period are: (1) to create and analyze human stem cell models of IDD; and (2) to create and analyze rodent models of IDD. Recognizing that sophisticated analysis of these models is required to elucidate pathogenesis and novel therapeutic approaches, the IDD Models Core uses extensive on-site research resources and leverages an extended network of complementary research cores on the UW- Madison campus to provide the infrastructure and skilled personnel that support integrated analysis at molecular, genomic, epigenomic, cellular, electrophysiological, and behavioral levels. Assembly of these diverse resources is designed to broaden the scope and depth of IDD model studies and to facilitate progress of IDD research from basic science to translational studies.