Genome Editing and Biological Effects Testing Section

3.摘要-基因组编辑和生物效应测试部分 水稻基因组编辑和生物效应测试部分（GE-BETS）的目标 编辑测试科的任务是向研究界提供一个集中的、可广泛使用的、收费服务的 用于在小鼠模型中测试基因组编辑递送试剂和基因组编辑工具的资源。GE-BETS 将开发新的方法和标准操作程序（SOP），并根据 研究需求。GE-BETS利用贝勒大学广泛的本地基因组编辑和测试经验 医学院（NMD）和赖斯大学，起源于基因敲除小鼠表型项目， 精准医学模型中心和NIH体细胞BCM-Rice小动物试验中心 基因组编辑程序。在该项目中，GE-BETS将开发用于测试基因组编辑试剂的管道 在基因组编辑报告和疾病模型，并为外部研究合作伙伴进行测试。目标1： 将在荧光报告小鼠中进行体细胞基因组编辑系统的测试。主要成果包括 生物分布、跨组织的编辑效率、细胞类型鉴定以及健康或毒性变化 从基因组编辑和传输系统。在目标2中，我们将确定 相关疾病模型中的基因组编辑试剂。我们将评估下一代的靶编辑和脱靶编辑 在靶组织中进行测序，以精确地量化基因组编辑的效率， 致病突变编辑效率将与预期基因的恢复或去除相关 产品这些研究将为新的治疗方法的可行性提供信息，并确定非预期的 基因组编辑的负面影响。在收费模式下巩固最先进的鼠标模型和专业知识， 服务的核心，将扩大获得高质量的测试数据，并加快新的 治疗学

3. ABSTRACT – GENOME EDITING AND BIOLOGICAL EFFECTS TESTING SECTION The goal of the Genome Editing and Biological Effects Testing Section (GE-BETS) of the BCM/Rice Genome Editing Testing Section is to provide to the research community a centralized, broadly accessible, fee-for-service resource for testing genome editing delivery reagents and genome editing tools in mouse models. The GE-BETS will develop new approaches and standard operating procedures (SOPs), as well as customize analyses based on research needs. GE-BETS capitalizes on extensive local genome editing and testing experience within Baylor College of Medicine (BCM) and Rice University, originating from the BCM Knockout Mouse Phenotyping Project, Center for Precision Medicine Models, and the BCM-Rice Small Animal Testing Center of the NIH Somatic Cell Genome Editing program. In this project, GE-BETS will develop pipelines for testing of genome editing reagents in genome editing reporter and disease models and perform testing for external research partners. In Aim 1, we will perform testing of somatic genome editing systems in fluorescent reporter mice. Major deliverables include biodistribution, editing efficiency across tissues, cell type identification, and changes in health or toxicity arising from genome editing and delivery systems. In Aim 2, we will determine the efficiency, specificity, and safety of genome editing reagents in relevant disease models. We will assess on- and off-target editing by next generation sequencing in the target tissue to precisely quantify the efficiency of genome editing in the context of a pathogenic mutation. Editing efficiency will be correlated with restoration or removal of the intended gene product. These studies will inform the feasibility of new therapeutic approaches, as well as identify unintended adverse effects of genome editing. Consolidating state of the art mouse models and expertise under a fee-for- service core, will broaden access to high quality testing data and accelerate the development of new therapeutics.