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

项目摘要--总体 基因组编辑系统有可能治愈一些最严重的人类疾病。然而，在那里 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 确定适用于特定组织的递送系统，评估总体效率和特异性 传递和基因组编辑系统，以及传递和基因组的有效性和安全性的评估 编辑技术。BCM-水稻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，并将把基因组编辑报告鼠品系存放到 一个公众可访问的储存库，供更广泛的科学界使用。我们的长期目标是 通过定义益处和定义，加速人类疾病体细胞基因组编辑的临床发展 新投递技术和编辑系统的风险。整个部分提供了以下声明 意义、要开展的工作的概要、可用于这项工作的资源的相关说明 在BCM和莱斯的项目，介绍了绩效指标和研究团队的资格，并说明了 与该提案相关的以前的专业知识和基础设施。总的来说，这些部分传达了 BCM-水稻作为新的体细胞基因编辑分析资源中心的建议选址。很明显 德克萨斯医疗中心(TMC)社区将为该项目带来重大好处，该项目将 也让TMC社区受益匪浅。

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.