Genome Editing Testing Core

点击翻译按钮获取中文摘要

Genome Editing Testing Core PROJECT SUMMARY / ABSTRACT The overarching goal of the Genome Editing Testing Core is to test the function of reagents and genome editing tools developed by investigators funded through other components of the Somatic Cell Genome Editing (SCGE) Program. The Genome Editing Testing Core will work directly with SCGE awardees and expand established standard operating procedures (SOPs) and develop new assays for detection of genome editing in wild-type and reporter nonhuman primates. The Genome Editing Testing Core will test the efficiency and specificity of editing and will assess the safety and immunogenicity of the genome editing technologies and delivery systems. The Genome Editing Testing Core has all of the essential facilities and capabilities required to test a range of viral and non-viral vectors developed by SCGE awardees. The Genome Editing Testing Core will contribute to the acceleration of the translation of genome editing technologies into treatments for human diseases through the following Specific Aims: (1) Evaluate genome editing in cells and tissues, (2) Address safety and the potential for inflammation and immune responses, and (3) Validation of reporter nonhuman primates. The Genome Editing Testing Core team has the expertise and flexibility to work with investigators to quantify any desired type of genome editing event in both rhesus macaques and marmosets (e.g., on-target, off-target, large rearrangements, duration of editing, cell specificity), and in any organ system or cell population. Paired with a robust Coordination Component and the Resource Core, and through the infrastructure of the UC Davis Genome Center, California National Primate Research Center, and Clinical and Translational Science Center, the Genome Editing Testing Core will deliver the data needed to comprehensively assess the promise of new technologies developed by SCGE awardees.