BCM-Rice resource for the analysis of somatic gene editing in mice

用于分析小鼠体细胞基因编辑的 BCM-Rice 资源

• 批准号: 10454900
• 负责人: Mary E Dickinson
• 金额: $ 67.57万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-09-18 至 2024-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10454900
• 关键词: Address; Animal Model; Award; Benefits and Risks; Clinical Trials; Communities; Commuting; Data; Deposition; Elements; Evaluation; Genes; Genome; Goals; Housing; Human Resources; Infrastructure; Knockout Mice; Leadership; Medical center; Modeling; Mouse Strains; Mus; Phenotype; Production; Reporter; Research; Research Personnel; Resources; Rice; Safety; Services; Site; Specificity; System; Technology; Testing; Texas; Tissues; Work; animal care; base; clinical development; collaborative environment; community center; efficacy evaluation; experience; gene therapy; genome editing; human disease; in vivo evaluation; member; mouse model; novel; programs; public repository; scientific organization; somatic cell gene editing; tool; translational approach

Project Summary – Overall Genome editing systems have the potential to cure some of the most severe human diseases. However, there are significant challenges that must be addressed before this technology can be applied in clinical trials including identification of appropriate delivery systems for specific tissues, evaluation of overall efficiency and specificity of delivery and genome editing systems, and assessment of efficacy and safety of both delivery and genome editing technologies. The BCM-Rice Resource Center for the Analysis of Somatic Gene Editing in Mice will create mouse model resources for in vivo testing of somatic genome editing tools developed by members of the Somatic Cell Genome Editing (SCGE) program, will assist researchers within the program with evaluation of genome editing strategies using these new models, and will deposit genome editing reporter mouse strains into a publically accessible repository for distribution to the wider scientific community. Our long term goal is to accelerate the clinical development of somatic genome editing for human disease by defining the benefits and risks of novel Delivery Technologies and Editing Systems. The Overall section provides a statement of significance, a synopsis of the work to be performed, relevant descriptions of the resources available for this project at BCM and Rice, a description of the qualifications of the PIs and research team and a description of previous expertise and infrastructure relevant to this proposal. Together, these sections convey the strengths of BCM-Rice as a proposed site for a new Resource Center for the Analysis of Somatic Gene Editing. It is clear that the Texas Medical Center (TMC) community will bring significant benefits to the project and this project will also strongly benefit the TMC community.

项目总结 – 总体 基因组编辑系统有潜力治愈一些最严重的人类疾病。然而，有 在将该技术应用于临床试验之前必须解决的重大挑战包括 确定适合特定组织的输送系统，评估整体效率和特异性 递送和基因组编辑系统的研究，以及递送和基因组的功效和安全性评估 编辑技术。 BCM-Rice 小鼠体细胞基因编辑分析资源中心将 创建小鼠模型资源，用于体内测试由成员开发的体细胞基因组编辑工具 体细胞基因组编辑（SCGE）计划将协助该计划内的研究人员评估 使用这些新模型的基因组编辑策略，并将基因组编辑报告小鼠品系存入 一个可公开访问的存储库，用于分发给更广泛的科学界。我们的长期目标是 通过明确其益处和作用，加速人类疾病体细胞基因组编辑的临床开发 新颖的交付技术和编辑系统的风险。总体部分提供了以下陈述 重要性、要执行的工作的概要、可用资源的相关描述 BCM 和 Rice 的项目，PI 和研究团队的资格描述以及 先前与本提案相关的专业知识和基础设施。这些部分共同传达了以下内容的优势： BCM-Rice 作为体细胞基因编辑分析新资源中心的拟议站点。很明显 德克萨斯医疗中心 (TMC) 社区将为该项目带来显着效益，该项目将 也使 TMC 社区受益匪浅。