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Development of Large Animal Reporter Systems for Testing Somatic Cell Genome Editing Tools (U24 Clinical Trial Not Allowed)
The purpose of this Funding Opportunity Announcement (FOA) is to create novel large animal reporter models for testing in vivo genome editing technologies. The deliverables from the initiative will be a set of large animal models (pigs and non-human primates), containing one or more reporter genes expressed in all cell types, that will allow facile and quantitative evaluation of genome editing in any cell type of interest. Within the Consortium, these models will be used to test new delivery systems and genome editing tools developed by accompanying components in planned Large Animal Testing Centers. The large animal models will allow investigators to optimize scale up of delivery systems and editing tools, which will be necessary for clinical trials in humans.
To maximize the potential of genome editing technology to treat as many diseases as possible, the SGCE program has been developed to accelerate the translation of genome editing technology into clinical applications. The key components of the SCGE program include: (1) better animal models for assessing genome editing in vivo (SCGE Animal Models, RFA-RM-18-012 and RFA-RM-18-013); (2) tools and assays to detect adverse consequences of genome editing in human cells (SCGE Biological Systems, RFA-RM-18-015); (3) new technologies to deliver genome editing machinery into disease-relevant cells and tissues in vivo (SCGE Delivery Technologies, RFA-RM-18-016); (4) novel genome editing and engineering systems (SCGE Editing Systems; RFA-RM-18-017); and (5) the SCGE Dissemination and Coordinating Center (SCGE DCC, RFA-RM-18-018). The SCGE DCC will make the results, tools and technologies developed under this program widely available to facilitate adoption for translation into clinical applications.
- Letter of Intent Due Date(s): March 3, 2018
- Application Due Date(s): April 3, 2018
RFA-RM-18-013 Expiration Date April 4, 2018
Application budgets should not exceed $500,000 direct costs per year in FY2018 - 2022 and need to reflect the actual needs of the proposed project but need to reflect the actual needs of the proposed project.
The maximum project period is 5 years (FY2018 - 2022).