Genome Editing Testing Core

基因组编辑测试核心 项目总结/摘要 基因组编辑测试核心的总体目标是测试试剂和基因组的功能 由研究人员开发的编辑工具，通过体细胞基因组编辑的其他组成部分资助 （SCGE）方案。基因组编辑测试核心将直接与SCGE获奖者合作， 建立标准操作程序（SOP），并开发新的检测基因组编辑的方法。 野生型和报告非人灵长类动物。基因组编辑测试核心将测试效率和 将评估基因组编辑技术的安全性和免疫原性， 运载系统。基因组编辑测试核心具有所需的所有基本设施和功能 测试SCGE获奖者开发的一系列病毒和非病毒载体。基因组编辑测试核心 将有助于加速基因组编辑技术转化为人类疾病的治疗方法。 通过以下具体目标来研究疾病：（1）评估细胞和组织中的基因组编辑，（2）解决 安全性以及炎症和免疫应答的可能性，和（3）非人报告基因的验证 灵长类动物基因组编辑测试核心团队拥有专业知识和灵活性，可以与研究人员合作， 定量恒河猴和绒猴中任何所需类型的基因组编辑事件（例如，达到目标， 脱靶、大重排、编辑持续时间、细胞特异性），以及在任何器官系统或细胞中 人口与强有力的协调部分和资源核心相配合，并通过 加州大学戴维斯分校基因组中心，加州国家灵长类动物研究中心，临床和 翻译科学中心，基因组编辑测试核心将提供所需的数据， 全面评估SCGE获奖者开发的新技术的前景。

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.