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.

项目总结