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

项目概要-总体 基因组编辑系统有可能治愈一些最严重的人类疾病。但 在这项技术应用于临床试验之前，必须解决的重大挑战包括 确定用于特定组织的适当递送系统，评价总体效率和特异性 递送和基因组编辑系统的有效性和安全性，以及递送和基因组编辑系统的有效性和安全性评估。 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，用于体内测试由美国国家生物技术委员会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，将协助研究人员在该计划与评估 使用这些新模型的基因组编辑策略，并将存款基因组编辑报告小鼠品系， 这是一个可供更广泛科学界使用的资料库。我们的长期目标是 通过定义益处，加速用于人类疾病的体细胞基因组编辑的临床开发， 新的交付技术和编辑系统的风险。总体部分提供了以下声明： 重要性，要执行的工作的概要，可用于此的资源的相关说明， 项目的主要研究人员和研究团队的资格说明，以及 与此提案相关的先前专业知识和基础设施。这些部分共同传达了 BCM-Rice作为一个新的体细胞基因编辑分析资源中心的建议网站。显然 德克萨斯州医疗中心（TMC）社区将为该项目带来重大利益，该项目将 这也给TMC社区带来了很大的好处。

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.