Genome Editing and Biological Effects Testing Component

基因组编辑和生物效应测试 项目总结/摘要 该中心的基因组编辑和生物效应测试组件的总体目标是 非人灵长类体细胞基因组编辑是为了测试开发的递送试剂和编辑工具 野生型、报告基因和疾病模型的研究者。进行的研究将解决效率， 基因组编辑技术的特异性和安全性，包括编辑活动的持续时间， 潜在的炎症和免疫原性。非人类灵长类动物将通过资源 协调部分将确保各项研究得到妥善管理和优先安排， 有效的数据收集、分析和共享。基因组编辑和生物效应测试组件 我将直接与研究人员合作，并利用已证明的能力来测试一系列病毒和非病毒 载体，并有助于加速体细胞基因组编辑技术转化为 治疗人类疾病。基因组编辑和生物效应测试组件将解决 以下具体目的：（1）在非人类中评估跨年龄组的体细胞基因组编辑 灵长类动物，和（2）评估生物效应，安全性，以及潜在的炎症和免疫反应， 非人类灵长类动物模型。基因组编辑和生物效应测试组件团队拥有 专业知识和灵活性与研究人员合作以量化任何期望类型的基因组编辑事件（例如， 中靶、脱靶、大重排、编辑持续时间、细胞特异性），并且在任何器官系统中，细胞 人口或年龄组。与强大的资源组件搭配，并通过UC的基础架构 戴维斯基因组中心，加州国家灵长类动物研究中心，临床和转化科学 基因组编辑和生物效应测试组件将提供所需的数据， 全面评估开发的新体细胞基因组编辑技术的前景， investigators.

Genome Editing and Biological Effects Testing PROJECT SUMMARY / ABSTRACT The overarching goal of the Genome Editing and Biological Effects Testing Component of the Center for Somatic Cell Genome Editing in Nonhuman Primates is to test delivery reagents and editing tools developed by investigators in wild-type, reporter, and disease models. Studies performed will address the efficiency, specificity, and safety of genome editing technologies, including the duration of editing activity, and the potential for inflammation and immunogenicity. Nonhuman primates will be provided through the Resource Component, and the Coordination Component will ensure studies are well managed and prioritized, with efficient data collection, analysis, and sharing. The Genome Editing and Biological Effects Testing Component will work directly with investigators and utilize demonstrated capabilities to test a range of viral and nonviral vectors and contribute to the acceleration of the translation of somatic cell genome editing technologies into treatments for human diseases. The Genome Editing and Biological Effects Testing Component will address the following Specific Aims: (1) Evaluate somatic cell genome editing across age groups in nonhuman primates, and (2) Assess biological effects, safety, and the potential for inflammation and immune responses in nonhuman primate models. The Genome Editing and Biological Effects Testing Component team has the expertise and flexibility to work with investigators to quantify any desired type of genome editing event (e.g., on-target, off-target, large rearrangements, duration of editing, cell specificity), and in any organ system, cell population, or age group. Paired with a robust Resource Component, and through the infrastructure of the UC Davis Genome Center, California National Primate Research Center, and Clinical and Translational Science Center, the Genome Editing and Biological Effects Testing Component will deliver the data needed to comprehensively assess the promise of new somatic cell genome editing technologies developed by investigators.