Scientific Research Resource Core

科研资源核心

• 批准号: 10261406
• 负责人: RHONDA BASSEL-DUBY
• 金额: $ 20.01万
• 依托单位: UT SOUTHWESTERN MEDICAL CENTER
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-09-15 至 2025-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10261406
• 关键词: Address; Affect; Blood; CRISPR/Cas technology; Cardiac Myocytes; Communities; Data; Disease; Duchenne muscular dystrophy; Dystrophin; Exons; Free Will; Funding; Genes; Genetic Diseases; Genome; Genomics; Goals; Guide RNA; Human; Individual; International; Learning; Link; Mediating; Modeling; Molecular; Muscle Cells; Muscular Dystrophies; Mutation; Myopathy; Patients; Process; Protocols documentation; RNA Sequences; Reagent; Research; Research Personnel; Research Support; Resources; Services; Site; Techniques; Technology; Therapeutic; Time; Training; Transcript; Update; Visit; biobank; clinical phenotype; design; efficacy evaluation; gene repair; genome editing; improved; in silico; induced pluripotent stem cell; mutant; next generation sequencing; novel; novel strategies; repair strategy; restoration; tool; training opportunity; web page; web site

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.

项目摘要 CRISPR/CAS9介导的基因组编辑的突破性技术提供了全新的 对于患有Duchenne等遗传疾病的患者的强大治疗潜力 肌肉营养不良（DMD）。自2015年加入Wellstone网络以来，UT Myoediting Core既是Wellstone Research的重要协调枢纽 UT西南部的小组（项目1和2），作为共享专业知识和资源的渠道 与更广泛的科学界。向前迈进，我们为这项共同研究的目标 资源核心是：（目标＃1）继续支持UT西南部的项目1和2 通过提供人IPSC的尖端技术，CRISPR/CAS9，Wellstone Center 基因组编辑和心肌细胞分化。 （目标＃2）帮助进步 通过提供患者衍生的DMD的DMD/BMD生物验证，全球肌肉营养不良 IPSC及其相应的CRISPR/CAS9校正分离株以及有关 供体患者的临床表型和基因组序列。 （目标＃3）帮助其他 调查人员通过自由分享我们的简化协议来充分利用这些资源 并在最佳实践和方法中提供培训（在现场或计算机上）。这些 来自其他井石中心以及一般的调查人员可以使用资源 整个科学界。