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Genome Editing and Biological Effects Testing Component

基因组编辑和生物效应测试组件

基本信息

批准号：
10773949
负责人：
DAVID J SEGAL
金额：
$ 65.19万
依托单位：
UNIVERSITY OF CALIFORNIA AT DAVIS
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：
2023-09-01 至 2028-08-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10773949
关键词：
Acceleration Address Adult Animals Antibodies B-Lymphocytes Biodistribution Biological Biological Assay Biological Markers Biopsy Brain California Cell physiology Cells Center for Translational Science Activities Clinical Chemistry Clinical Sciences Collaborations Communities DNA Data Data Collection Disease Disease model Ear Ensure Equity Evaluation Event Eye Female Frequencies Genes Genome Genomic DNA Goals Gonadal structure Heart Hematology Hematopoietic System Histologic Homeostasis Immune response In Vitro Inflammation Infrastructure Kidney Link Lipids Liver Lung Measurement Metabolic Methods Modeling Muscle Mutation Natural Immunity Non-Viral Vector Population Group Positron-Emission Tomography Primates Procedures Process Proteins Quality Control Reagent Reporter Reproducibility Research Research Design Research Personnel Resolution Resources Safety Sex Differences Site Somatic Cell Specificity Standardization System T cell response T-Lymphocyte Techniques Technology Testing Tissues Translations Viral Vector Visualization Work X-Ray Computed Tomography adaptive immunity age group base editor body system bone cell type comparative delivery vehicle design disease phenotype fetal flexibility genome editing human disease immunogenicity in vivo in vivo imaging insertion/deletion mutation male nonhuman primate off-target site optical imaging organizational structure programs quality assurance shared database single-cell RNA sequencing somatic cell gene editing tool

项目摘要

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.

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.