Scientific Research Resource Core

项目摘要 CRISPR/Cas9介导的突破性基因组编辑技术提供了一种全新的 对患有杜兴等遗传病的患者具有强大的治疗潜力 肌营养不良症(DMD)。自2015年加入威尔斯通网络以来，德克萨斯大学西南部 Myoediting Core一直是Wellstone研究的重要协调中心 德克萨斯大学西南分校的小组(项目1和2)，并作为分享专业知识和资源的渠道 与更广泛的科学界合作。展望未来，我们对这项共享研究的目标 资源核心是：(目标1)继续支持德克萨斯大学西南部的项目1和2 通过提供人类IPSCs的尖端技术，CRISPR/CAS9 基因组编辑和心肌细胞分化。(目标2)帮助推进对 通过提供患者来源的DMD/BMD生物信息库，全球肌营养不良症 IPSCs及其相应的CRISPR/CAS9更正的分离物以及关于 供体患者的临床表型和基因组序列。(目标3)帮助他人 调查人员通过自由共享我们简化的协议来充分利用这些资源 并提供最佳做法和方法方面的培训(现场培训或硅胶培训)。这些 其他韦尔斯通中心的调查人员以及将军都可以获得资源 整个科学界。

Project Summary The breakthrough technology of CRISPR/Cas9-mediated genomic editing offers an entirely new and powerful therapeutic potential for patients suffering from genetic diseases like Duchenne Muscular Dystrophy (DMD). Since joining the Wellstone Network in 2015, the UT Southwestern Myoediting Core has served both as an essential coordinating hub for the Wellstone research groups (Projects 1 & 2) at UT Southwestern and as a conduit for sharing expertise and resources with the broader scientific community. Moving forward, our goals for this Shared Research Resource Core are: (Aim #1) To continue to support Projects 1 and 2 of the UT Southwestern Wellstone Center by providing the cutting-edge technologies of human iPSCs, CRISPR/Cas9 genomic editing, and cardiomyocyte differentiation. (Aim #2) To help advance the study of muscular dystrophy worldwide by providing a DMD/BMD Biorepository of patient-derived DMD iPSCs and their corresponding CRISPR/Cas9-corrected isolates along with information regarding the clinical phenotype and genomic sequence of the donor patient. (Aim #3) To help other investigators take full advantage of these resources by freely sharing our streamlined protocols and providing training (either on site, or in silico) in best practices and approaches. These resources are available to investigators from other Wellstone Centers as well as the general scientific community at large.