Testing Centers for Development of Somatic Cell Genome Editing in Model Organisms (U42) (Clinical Trials Not Allowed)

The purpose of this Funding Opportunity Announcement (FOA) is to support Testing Centers for Somatic Cell Genome Editing in specified major mammalian animal model species (rodents, pigs, and non-human primates). Each Center will provide resources and testing services to the growing community developing new genome editing technologies and conducting preclinical testing based upon these technologies for therapeutics of human diseases. The Testing Centers will work with NIH funded investigators to assess the efficacy and safety of in vivo genome editing and delivery technologies, determine genome editing thresholds for specific diseases associated with minimal off-target effects, and ascertain feasibility parameters in a model system. This data will be important in determining if a treatment strategy using a specific set of tools is effective and has an acceptable safety profile, or if the efficacy and specificity of editing should be significantly improved. Even though each Testing Center will work on a limited number of focused projects, it is expected that the program and pipeline(s) developed will maintain multifaceted research activities to build core model systems that can be adjusted as required to accommodate a broad spectrum of diseases as well as provide fee-for-service to the wider biomedical community on a national basis.
Categories: Health.