BCM/RICE GENOME EDITING TESTING CENTER

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1. ABSTRACT – OVERALL Somatic cell genome editing holds tremendous promise as a potential cure for the most severe of human diseases. However, many new technologies do not progress into humans because of insufficient preclinical data in animal models. Mice are particularly important early in the preclinical development pipeline because of their genetic tractability and similarities to humans in development, physiology, metabolism and disease. Fluorescent reporter mice are a powerful tool for assessing genome editing efficacy across multiple organ systems and make it possible to quantify editing events down to single cell resolution. Mouse disease models harboring pathogenic mutations can be used to determine the nature of on- and off-target editing events that may occur, the safety of a genome editing approach, and the genome editing threshold required for disease amelioration. Solving the challenges of somatic genome editing is a formidable interdisciplinary effort requiring diverse expertise ranging from molecular biology and bioengineering to pathology and immunology. Thus, it is critical to have resources for animal model testing that can bring these components together in one place. We will leverage the expertise and resources of our existing large-scale, NIH-funded projects centered around mouse modeling, including our current BCM-Rice Small Animal Testing Center (SATC) of the Somatic Cell Genome Editing (SCGE) program, to form the BCM/Rice Genome Editing Testing Center (GETC). The vision of the GETC is to offer high-quality mouse resources and robust somatic genome editing testing pipelines that can support researchers developing new genome editing technologies and conducting preclinical tests with these novel tools. Our goal is to use wild- type, genome editing reporter, and human disease model mouse lines to evaluate (1) the efficacy, tissue specificity, and safety of genome editing technologies and delivery systems and (2) disease gene editing approaches and genome editing thresholds required to ameliorate specific diseases. By centralizing mouse resources and testing fee-for-services within an environment harboring a supportive and experienced research team, the GETC can ensure the highest experimental standards, rigor, and reproducibility. We expect the GETC will make a broad and sustained impact by supporting the development of new genome editing technologies and accelerating their effective and safe deployment to the clinic.