Project Summary – Overall Genome editing systems have the potential to cure some of the most severe human diseases. However, there are significant challenges that must be addressed before this technology can be applied in clinical trials including identification of appropriate delivery systems for specific tissues, evaluation of overall efficiency and specificity of delivery and genome editing systems, and assessment of efficacy and safety of both delivery and genome editing technologies. The BCM-Rice Resource Center for the Analysis of Somatic Gene Editing in Mice will create mouse model resources for in vivo testing of somatic genome editing tools developed by members of the Somatic Cell Genome Editing (SCGE) program, will assist researchers within the program with evaluation of genome editing strategies using these new models, and will deposit genome editing reporter mouse strains into a publically accessible repository for distribution to the wider scientific community. Our long term goal is to accelerate the clinical development of somatic genome editing for human disease by defining the benefits and risks of novel Delivery Technologies and Editing Systems. The Overall section provides a statement of significance, a synopsis of the work to be performed, relevant descriptions of the resources available for this project at BCM and Rice, a description of the qualifications of the PIs and research team and a description of previous expertise and infrastructure relevant to this proposal. Together, these sections convey the strengths of BCM-Rice as a proposed site for a new Resource Center for the Analysis of Somatic Gene Editing. It is clear that the Texas Medical Center (TMC) community will bring significant benefits to the project and this project will also strongly benefit the TMC community.

Genome editing systems have the potential to cure some of the most severe human diseases. However, there are significant efficacy and safety issues that must be addressed before this technology can be applied in clinical trials. The BCM-Rice Resource Center for the Analysis of Somatic Gene Editing in Mice will create mouse reporter models for testing genome editing technologies, and to use these animal models to test genome editing delivery technologies and new genome editors developed by other Somatic Cell Genome Editing program members.