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Genome Editing and Biological Effects Testing Section

基因组编辑及生物效应检测组

基本信息

批准号：
10773478
负责人：
William Raymond Lagor
金额：
$ 47.07万
依托单位：
BAYLOR COLLEGE OF MEDICINE
依托单位国家：
美国
项目类别：
财政年份：
2023
资助国家：
美国
起止时间：
2023-12-01 至 2028-11-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10773478
关键词：
Acceleration Adverse effects Alleles Animal Testing Animals Biodistribution Biological Cells Chemistry Chromosomal translocation Communities Consult Data Development Disease Disease model Enzyme-Linked Immunosorbent Assay Event Excision Fee-for-Service Plans Flow Cytometry Germ Cells Goals Health Histologic Histopathology Immune response Immunophenotyping Knockout Mice Medicine Methods Modeling Monitor Mus Mutation Organ Pathogenicity Phase Phenotype Preclinical Testing Procedures Protein Secretion Reagent Reporter Reporting Research Research Personnel Resources Rice Safety Serum Specificity System Testing Time Tissue Harvesting Tissues Toxic effect United States National Institutes of Health Universities Western Blotting Work automated image analysis base editing cell type central database college experience experimental study gene product genome editing insertion/deletion mutation interest mouse model next generation sequencing novel strategies novel therapeutic intervention novel therapeutics precision medicine prime editing programs repaired restoration somatic cell gene editing tool vector genome

项目摘要

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.

3.摘要-基因组编辑和生物效应测试部分 BCM/水稻基因组编辑和生物效应测试科(GE-BETTS)的目标编辑测试科将为研究社区提供一个集中化的、广泛访问的、收费的服务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。通用电气的赌注将开发新的方法和标准操作程序(SOP)，并基于以下内容定制分析关于研究的需要。GE-Bets利用贝勒公司丰富的本地基因组编辑和测试经验医学院(BCM)和莱斯大学，起源于BCM基因敲除老鼠表型项目，精密医学模型中心和美国国立卫生研究院体细胞BCM-大米小动物实验中心基因组编辑程序。在这个项目中，GE-BETTS将开发用于测试基因组编辑试剂的管道在基因组编辑、记者和疾病模型方面，并为外部研究合作伙伴进行测试。在目标1中，我们将在荧光报告鼠身上进行体细胞基因组编辑系统的测试。主要交付成果包括生物分布、跨组织的编辑效率、细胞类型识别以及健康或毒性引起的变化来自基因组编辑和传输系统。在目标2中，我们将确定药物的有效性、特异性和安全性。相关疾病模型中的基因组编辑试剂。我们将评估下一代的目标上和目标外的编辑在靶组织中进行测序以精确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移除相关产品。这些研究将为新的治疗方法的可行性提供信息，并识别非预期的基因组编辑的不利影响。将最先进的鼠标模型和专业知识整合到按需付费的服务核心，将拓宽获取高质量测试数据的途径，并加速开发新的治疗学。