Genome Editing and Biological Effects Testing Section

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3. ABSTRACT – GENOME EDITING AND BIOLOGICAL EFFECTS TESTING SECTION The goal of the Genome Editing and Biological Effects Testing Section (GE-BETS) of the BCM/Rice Genome Editing Testing Section is to provide to the research community a centralized, broadly accessible, fee-for-service resource for testing genome editing delivery reagents and genome editing tools in mouse models. The GE-BETS will develop new approaches and standard operating procedures (SOPs), as well as customize analyses based on research needs. GE-BETS capitalizes on extensive local genome editing and testing experience within Baylor College of Medicine (BCM) and Rice University, originating from the BCM Knockout Mouse Phenotyping Project, Center for Precision Medicine Models, and the BCM-Rice Small Animal Testing Center of the NIH Somatic Cell Genome Editing program. In this project, GE-BETS will develop pipelines for testing of genome editing reagents in genome editing reporter and disease models and perform testing for external research partners. In Aim 1, we will perform testing of somatic genome editing systems in fluorescent reporter mice. Major deliverables include biodistribution, editing efficiency across tissues, cell type identification, and changes in health or toxicity arising from genome editing and delivery systems. In Aim 2, we will determine the efficiency, specificity, and safety of genome editing reagents in relevant disease models. We will assess on- and off-target editing by next generation sequencing in the target tissue to precisely quantify the efficiency of genome editing in the context of a pathogenic mutation. Editing efficiency will be correlated with restoration or removal of the intended gene product. These studies will inform the feasibility of new therapeutic approaches, as well as identify unintended adverse effects of genome editing. Consolidating state of the art mouse models and expertise under a fee-for- service core, will broaden access to high quality testing data and accelerate the development of new therapeutics.