Training Core

项目总结/摘要 CRISPR/Cas9介导的基因组编辑的技术和产生诱导的表达的能力， 来自患者血液样本的多能干细胞（iPSC）已经将医学置于一个危险的边缘。 这将是我们治疗甚至治愈一系列基于基因组的疾病的能力的革命。超过 在过去的四年里，威尔士大学的威尔斯通肌肉营养不良合作研究中心 得克萨斯州西南医学中心（现在的UTSW韦尔斯通MDSRC）已经取得了巨大的进步， 致力于将这项技术转化为有益于杜氏肌营养不良症患者的技术， 最终导致其他形式的肌肉萎缩症。训练核心的使命 影响力，因为它对培养新一代临床医生，医生科学家和基础科学家至关重要 他们能够将这些不断发展的技术应用于肌肉萎缩症患者的服务， 社区培训核心的目标是改善井石的教育环境 网络，以招募，培训和保持下一代变革调查员的重点 应对肌营养不良症的挑战。我们的方法侧重于五个要素：（目标1） 改善UTSW Wellstone中心的培训环境，促进学员的职业发展 进步，（目标2）招募新学员进行肌肉萎缩症研究，（目标3）指导 （目标4）为学员的下一步职业生涯提供资金支持， （目标5）组织培训活动， 整个肌肉萎缩症研究界。为了实现这些目标，我们建立在 在过去四年的资助中，成功开发了一个项目， 充分利用了UTSW现有培训项目的优势。

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. Over the past four years the Wellstone Muscular Dystrophy Cooperative Research Center at the University of Texas Southwestern Medical Center (now the UTSW Wellstone MDSRC) has made tremendous strides toward the translation of this technology to benefit patients with Duchenne muscular dystrophy, and ultimately, other forms of muscular dystrophy. The mission of the Training Core has been just as impactful as it is essential to train a new generation of clinicians, physician-scientists, and basic scientists who are 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 of the Wellstone network in order to recruit, train, and maintain the next generation of transformative investigators focused on addressing the challenges of muscular dystrophy. Our approach focuses on five elements: (Aim 1) To enhance the training environment of the UTSW Wellstone Center and promote trainee career advancement, (Aim 2) To recruit new trainees to muscular dystrophy research, (Aim 3) To mentor trainees in the process of obtaining funding support for the next step in their careers, (Aim 4) To organize research meetings involving Wellstone trainees, and (Aim 5) To organize training activities that benefit the overall muscular dystrophy research community. To accomplish these goals, we’ve built on the very successful program developed over the past four years of funding, that has both complemented and leveraged the strengths of existing training programs here at UTSW.