Scientific Research Resource Core

科研资源核心

• 批准号: 10473540
• 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/10473540
• 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; induced pluripotent stem cell derived cardiomyocytes; 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.

项目概要 CRISPR/Cas9介导的基因组编辑的突破性技术提供了全新的方法 对于患有 Duchenne 等遗传性疾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 Wellstone 网络以来，UT Southwestern Myoediting Core 既是 Wellstone 研究的重要协调中心 UT Southwestern 的小组（项目 1 和 2），并作为共享专业知识和资源的渠道 与更广泛的科学界。展望未来，我们的共享研究目标 资源核心是：（目标#1）继续支持 UT 西南大学的项目 1 和 2 Wellstone中心提供人类iPSC、CRISPR/Cas9的尖端技术 基因组编辑和心肌细胞分化。 （目标#2）帮助推进研究 通过提供源自患者的 DMD 的 DMD/BMD 生物存储库，在全球范围内防治肌营养不良症 iPSC 及其相应的 CRISPR/Cas9 校正分离株以及有关信息 供体患者的临床表型和基因组序列。 （目标#3）帮助他人 研究人员通过免费分享我们简化的方案来充分利用这些资源 提供最佳实践和方法的培训（现场或计算机）。这些 其他 Wellstone 中心以及一般机构的调查人员均可使用资源 整个科学界。