Resource Component

资源 项目摘要/摘要 中国体细胞基因组编辑中心资源中心的总体目标 非人灵长类动物是为非人灵长类动物(野生型、报告型、疾病模型)提供数量和 为满足调查人员的需要所必需的素质。资源组件嵌入在 加州国家灵长类研究中心提供的成熟、全面的基础设施 标准操作程序(SOP)和质量保证/质量控制(QA/QC)职能范围 确保在最高质量标准下达到目标和目的。非人灵长类动物将是 由检测中心使用已建立的SOP进行筛选和选择，用于在基因组中进行的研究 编辑和生物效应测试组件，将评估效率、特异性、安全性和 所提议技术的免疫原性。协调部分将确保 在测试中心的所有组件中都采用这种方法。资源组件将加速翻译 从非人灵长类动物研究到人类治疗的体细胞基因组编辑技术 疾病通过以下具体目标：(1)提供高质量的跨年龄段的动物用于躯体 细胞基因组编辑研究，以及(2)筛选和选择非人类灵长类动物以评估体细胞基因组 编辑技术。资源部分将确保调查人员具备以下素质和数量 非人灵长类在所有年龄段都是必要的。男性和女性将被纳入研究队列，以及 选择样本量以确保数据具有统计学意义，以及严谨性和可重复性。与 基因组编辑和生物效应测试部分，并在基础设施的背景下 加州大学戴维斯分校国家灵长类研究中心，资源部分将提供动物和 进行必要的程序来全面评估新的体细胞基因组编辑的前景 未来治疗人类疾病的技术。

Resource PROJECT SUMMARY / ABSTRACT The overarching goal of the Resource Component of the Center for Somatic Cell Genome Editing in Nonhuman Primates is to provide nonhuman primates (wild-type, reporter, disease models) in the quantity and quality necessary to meet the needs of investigators. The Resource Component is embedded within the established, comprehensive infrastructure provided by the California National Primate Research Center with a range of standard operating procedures (SOPs) and Quality Assurance/Quality Control (QA/QC) functions to ensure that the goals and objectives are met under the highest quality standards. Nonhuman primates will be screened and selected by the Testing Center using established SOPs for studies conducted in the Genome Editing and Biological Effects Testing Component that will evaluate efficiency, specificity, safety, and immunogenicity of the technologies proposed. The Coordination Component will ensure an integrated approach across all components of the Testing Center. The Resource Component will accelerate translation of somatic cell genome editing technologies from studies in nonhuman primates into treatments for human diseases through the following Specific Aims: (1) Provide high quality animals across age groups for somatic cell genome editing studies, and (2) Screen and select nonhuman primates to evaluate somatic cell genome editing technologies. The Resource Component will ensure investigators have the quality and quantity of nonhuman primates necessary across all age groups. Males and females will be included in study cohorts, and sample size selected to ensure statistically significant data, as well as rigor and reproducibility. Paired with the Genome Editing and Biological Effects Testing Component, and within the context of the infrastructure of the UC Davis California National Primate Research Center, the Resource Component will deliver the animals and conduct the procedures necessary to comprehensively assess the promise of new somatic cell genome editing technologies for the future treatment of human disease.