Genome Editing and Biological Effects Testing: Somatic Cell Gene Editing Testing

PROJECT SUMMARY: GENOME EDITING TESTING SECTION This project addresses an unmet need for essential services related somatic cell genome editing. This project will provide models that recapitulate human conditions and harbor human target sequences. Further, this project will provide testing services to evaluate safety and efficacy of potential therapeutic strategies. The Coordination Section provides for central management, coordination, and communication for the Resource and Testing Sections. This core ensures that data will be preserved and appropriately communicated. These responsibilities also include communication with Center Users, the Program Steering Committees, and various collaborators. The Resource Section will be to produce, import, cryopreserve, validate, and supply unique swine models for testing somatic cell genome editing reagents. The Resource Section will maintain breeding colonies and produce cohorts of experimental animals. These novel swine models will provide unique opportunities to translate basic science to clinical applications. The Testing Section will be responsible for creating, refining, and performing detailed standard operating procedures (SOPs) and comprehensive reports for testing new genome editing technologies. This section will provide for robust, standardized, and comprehensive analysis of potential therapies in large animals to facilitate in translation of basic research into clinical applications.