The Jackson Laboratory Gene Editing Testing Center (JAX-GETC)

项目概要 JAX基因组编辑小鼠测试中心（JAX-GEMTC）旨在利用快速融合的技术， 基因组编辑技术的进步和基因治疗平台的成熟， 基因疾病治疗的突破为了完成这一使命，JAX-GEMTC将建立新的 报告小鼠线平台，用于编辑活动的稳健和灵敏的检测，并使用这些模型， 支持新的递送技术和编辑器的体内评价。该中心利用独特的 JAX的专业知识和无与伦比的小鼠遗传资源，以及80年的小鼠生物学历史 research.我们组建了一支才华横溢的多功能团队， 利用JAX机构管道和资源。三个研究组成部分-动物模型 生产单元、基因编辑测试核心、动物模型资源核心--紧密配合 由中心领导，主要建立在现有的研究和管理团队。中心 通过合作补充这些渠道，增加独特的资源和技能，以加快我们的 实施和扩大我们的影响的潜在范围。JAX-GEMTC的总体目标是：1） 开发小鼠模型资源，包括一组报告模型，以有力地监测基因组编辑 活性和细胞类型特异性。2)建立一个多学科专家小组， 可扩展的平台，以支持基因组编辑组件的交付和编辑结果的分析。3)到 通过部署系统，参与体细胞基因组编辑（SCGE）联盟并为其做出贡献， 共享数据和动物资源。4)为我们未来的合作扩展制定框架， 程序，利用我们的专业知识和我们的小鼠模型生成和体内的可扩展性， 操纵平台。这些目标的成功完成将推动SCGE计划的目标， 旨在改进基因组编辑技术，以加速将该技术转化为临床应用。 应用和最大限度地发挥潜力，治疗尽可能多的疾病。

PROJECT SUMMARY OVERALL SECTION The JAX Genome Editing Mouse Testing Center (JAX-GEMTC) aims to capitalize on the convergence of rapid advances in genome editing technology and the maturity of gene therapy platforms to achieve fundamental breakthroughs in the treatment of genetic disease. To accomplish this mission, the JAX-GEMTC will build novel reporter mouse line platforms for robust and sensitive detection of editing activity, and use these models to support the in vivo evaluation of new delivery technologies and editors. This Center draws upon the unique expertise and unparalleled mouse genetic resources at JAX, fortified by an 80-year history of mouse biology research. We have assembled a talented, multi-functional team organized into discrete components that leverage JAX institutional pipelines and resources. The three research components—the Animal Model Production Unit, the Gene Editing Testing Core, and the Animal Model Resource Core—are tightly coordinated by the Center leadership, and build primarily upon existing research and management teams. The Center supplements these pipelines with collaborations that add unique resources and skills to accelerate our implementation and expand the potential scope of our impact. The overall Aims of the JAX-GEMTC are: 1) To develop a mouse model resource, including a panel of reporter models, to robustly monitor genome editing activity and cell-type specificity in vivo. 2) To establish a multi-disciplinary team of experts and to develop a scalable platform to support the delivery of genome editing components and analysis of editing outcomes. 3) To engage with and contribute to the Somatic Cell Genome Editing (SCGE) consortium by deploying systems to share both data and animal resources. 4) To develop a framework for future collaborative expansion of our program, taking advantage of our expertise and the scalability of our mouse model generation and in vivo manipulation platforms. The successful completion of these aims will fuel the goals of the SCGE program, which aims to improve genome editing technologies to accelerate the translation of this technology into clinical applications and maximize the potential to treat as many diseases as possible.