PROJECT SUMMARY OVERALL SECTION The JAX Genome Editing Mouse Testing Center (JAX-GEMTC) aims to capitalize on the convergence of rapid advances in genome editing technology and the maturity of gene therapy platforms to achieve fundamental breakthroughs in the treatment of genetic disease. To accomplish this mission, the JAX-GEMTC will build novel reporter mouse line platforms for robust and sensitive detection of editing activity, and use these models to support the in vivo evaluation of new delivery technologies and editors. This Center draws upon the unique expertise and unparalleled mouse genetic resources at JAX, fortified by an 80-year history of mouse biology research. We have assembled a talented, multi-functional team organized into discrete components that leverage JAX institutional pipelines and resources. The three research components—the Animal Model Production Unit, the Gene Editing Testing Core, and the Animal Model Resource Core—are tightly coordinated by the Center leadership, and build primarily upon existing research and management teams. The Center supplements these pipelines with collaborations that add unique resources and skills to accelerate our implementation and expand the potential scope of our impact. The overall Aims of the JAX-GEMTC are: 1) To develop a mouse model resource, including a panel of reporter models, to robustly monitor genome editing activity and cell-type specificity in vivo. 2) To establish a multi-disciplinary team of experts and to develop a scalable platform to support the delivery of genome editing components and analysis of editing outcomes. 3) To engage with and contribute to the Somatic Cell Genome Editing (SCGE) consortium by deploying systems to share both data and animal resources. 4) To develop a framework for future collaborative expansion of our program, taking advantage of our expertise and the scalability of our mouse model generation and in vivo manipulation platforms. The successful completion of these aims will fuel the goals of the SCGE program, which aims to improve genome editing technologies to accelerate the translation of this technology into clinical applications and maximize the potential to treat as many diseases as possible.

PROJECT NARRATIVE OVERALL SECTION The revolution in gene editing technology promises to transform the development of therapeutics to treat human disease. As part of the Somatic Cell Genome Editing consortium, the goal of this project is to build mouse resources and provide an animal model testing platform to support the optimization of novel genome editing technologies for future translational applications.