Genome Editing Testing Core

基因组编辑测试核心 项目摘要/摘要 基因组编辑测试核心的首要目标是测试试剂和基因组的功能 由研究人员通过体细胞基因组编辑的其他组成部分资助开发的编辑工具 (SCGE)计划。基因组编辑测试核心将直接与SCGE获奖者合作并扩展 建立标准操作程序(SOP)并开发检测基因组编辑的新方法 野生型和记者非人灵长类动物。基因组编辑测试核心将测试效率和 并将评估基因组编辑技术的安全性和免疫原性，以及 传输系统。基因组编辑测试核心具有所需的所有基本设施和功能 测试由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 将有助于加速将基因组编辑技术转化为人类的治疗方法 疾病通过以下具体目标：(1)评估细胞和组织中的基因组编辑，(2)解决 安全性和炎症和免疫反应的可能性，以及(3)非人类报告的有效性 灵长类动物。基因组编辑测试核心团队拥有与调查人员合作的专业知识和灵活性 量化恒河猴和绒猴中任何期望类型的基因组编辑事件(例如，在目标上， 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。与强大的协调组件和资源核心配合使用，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.