Training Core

项目摘要/摘要 CRISPR/Cas9介导的基因组编辑技术及其诱导产生能力 从患者血液样本中提取的多能干细胞(IPSCs)使药物处于 我们治疗、甚至治愈广泛的基于基因组的疾病的能力发生了革命。完毕 在过去的四年里，华盛顿大学韦尔斯通肌肉营养不良合作研究中心 德克萨斯西南医学中心(现在的UTSW Wellstone MDSRC)已经取得了巨大的进步 将这项技术转化为Duchenne肌营养不良症患者，并 最终，其他形式的肌肉营养不良。培训核心的使命就是 因为培养新一代临床医生、内科科学家和基础科学家是必要的 谁能够将这些不断发展的技术应用于肌营养不良症患者和他们的 社区。培训核心的目标是改善韦尔斯通的教育环境 网络，以招募、培训和保持专注于变革的下一代调查人员 关于解决肌营养不良症的挑战。我们的方法侧重于五个要素：(目标1) 改善威斯通大学韦尔斯通中心的培训环境，促进学员职业生涯 发展，(目标2)招募新的受训人员从事肌营养不良研究，(目标3)指导 正在为其职业生涯的下一步获得资金支持的受训人员，(目标4)组织 参加由韦尔斯通受训人员参加的研究会议，并(目标5)组织有益的培训活动 整个肌营养不良症研究社区。为了实现这些目标，我们建立在非常 在过去四年的资助中制定了成功的计划，这既补充了 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.