Scientific Research Resource Core

项目摘要 CRISPR/Cas9介导的基因组编辑的突破性技术提供了一种全新的 对杜氏症等遗传病患者具有强大的治疗潜力 肌营养不良症（DMD）。自2015年加入Wellstone网络以来，UT西南 Myoediting Core既是Wellstone研究的重要协调中心， 小组（项目1和2）在UT西南和作为一个管道，分享专业知识和资源 更广泛的科学界。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UT西南的项目1和2 Wellstone中心通过提供人类iPSCs的尖端技术，CRISPR/Cas9 基因组编辑和心肌细胞分化。(Aim#2）帮助推进研究 通过提供患者源性DMD的DMD/BMD生物储存库， iPSC及其相应的CRISPR/Cas9校正的分离物沿着有关于 供体患者的临床表型和基因组序列。(Aim#3帮助他人 调查人员充分利用这些资源，免费共享我们的简化协议 提供最佳做法和方法方面的培训（现场或计算机模拟）。这些 资源可供调查人员从其他韦尔斯通中心以及一般 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.