Training Core

培训核心

• 批准号: 8975882
• 负责人: Jaya R Trivedi
• 金额: $ 10.8万
• 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 2020-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8975882
• 关键词: Address; Blood; Clustered Regularly Interspaced Short Palindromic Repeats; Collaborations; Communication; Communities; Complement; Disease; Duchenne muscular dystrophy; Environment; Financial Support; Fostering; Generations; Genome; Mediating; Medical center; Medicine; Mentors; Muscular Dystrophies; Patients; Physicians; Recruitment Activity; Research; Research Personnel; Sampling; Scientist; Services; Site; Techniques; Technology; Texas; Training; Training Programs; Translations; Universities; abstracting; base; genome editing; induced pluripotent stem cell; next generation

Project Summary/Abstract The techniques of CRISPR/Cas9-mediated genomic editing and the ability to generate induced pluripotent stem cells (iPSCs) from a sample of a patient's blood have placed medicine on the brink of a revolution in our ability to treat, and perhaps even cure, a broad range of genome-based diseases. The proposed Wellstone Muscular Dystrophy Cooperative Research Center at the University of Texas Southwestern Medical Center (UTSW Wellstone MDCRC) is uniquely poised to begin translation of this technology to benefit patients with Duchenne muscular dystrophy, and ultimately, other forms of muscular dystrophy. For this to become a reality, it is essential to train a new generation of clinicians, physician-scientists, and basic scientists who will be able to apply these evolving technologies in the service of muscular dystrophy patients and their community. The objective of the Training Core is to enhance the educational environment in order to recruit, train, and maintain the next generation of transformative investigators focused on addressing the challenges of muscular dystrophy. Our approach will be three fold: (1) To provide mentoring and financial support for two competitive, pilot and feasibility projects each year focused on advancing our understanding or treatment of any of the diverse forms of muscular dystrophy; (2) To develop and coordinate educational opportunities that will benefit all investigators and trainees associated with the Wellstone Center by both complementing and leveraging the strengths of existing training programs here at UTSW; (3) To foster avenues of interaction that will facilitate communication, collaboration, and synergy between the investigators at the UTSW Wellstone Center and those at other Wellston MDCRC network sites.

项目摘要/摘要 CRISPR/CAS9介导的基因组编辑的技术和产生诱导的能力 来自患者血液样本的多能干细胞（IPSC）已将药物放在A的边缘 我们治疗甚至可以治愈的能力的革命，这些革命是基于基因组的广泛疾病。这 提议的井石肌营养不良合作研究中心，德克萨斯大学 西南医学中心（UTSW Wellstone MDCRC）独特地准备开始翻译此事 使Duchenne肌营养不良症患者以及其他形式的技术受益的技术 肌肉营养不良。要使这成为现实，培训新一代临床医生至关重要 医师科学家和基本科学家将能够将这些不断发展的技术应用于 服务肌肉营养不良的患者及其社区。训练核心的目的是 增强教育环境以招募，训练和维护下一代 变革性研究人员致力于应对肌肉营养不良的挑战。我们的 方法将为三倍：（1）为两个竞争性的飞行员提供指导和财政支持 每年的可行性项目都集中于我们对任何一项的理解或治疗 多种形式的肌肉营养不良； （2）开发和协调教育机会 通过补充和 利用UTSW现有培训计划的优势； （3）促进 互动将促进调查人员之间的沟通，协作和协同作用 UTSW Wellstone中心以及其他Wellston MDCRC网站的中心。