Training Program in Stem Cell Translational Medicine for Neurological Disorders

项目摘要 干细胞医学有望彻底改变人类疾病和损伤的治疗，并已捕获 科学界和公众的希望。也许干细胞的潜力 治疗人类疾病和损伤比治疗神经系统疾病更有前途。从“板凳”到“ 床旁”仍然是研究人员的一个相对较新的机会，并为培养研究生提供了新的挑战 学生从历史上看，神经科学博士生接受的培训是了解基本的生物学知识， 大脑如何运作和引导行为、学习和记忆、运动的机制，以及 找出失调并导致神经系统疾病的过程。事实上，生物学 许多神经系统疾病的机制和原因已经确定，为神经系统疾病的治疗铺平了道路。 产生转基因动物和模型系统，以研究神经系统疾病。关键是，这些 这些进展，加上干细胞生物学的出现，开辟了一条治疗神经系统疾病的道路， 干细胞衍生的细胞群体的移植或开发干细胞衍生的细胞中的治疗策略 模型这是一条需要基础科学之外的训练的道路。因此，我们的培训目标 在干细胞转化医学神经疾病计划是提供机会， 博士前学员进行翻译“板凳”研究，可以治疗人类神经系统疾病 和伤害，在精神上的NIH的愿景转化医学。此外，为了应对挑战， 随着这一新领域的出现，我们为实习生提供了临床方面的机会， 他们正在研究的疾病。这些机会使得能够识别具有最大 与人类疾病的相关性，为临床前安全性和有效性测试提供信息，并定义临床前结果 与临床指标平行的指标。此外，该计划使学员熟悉的复杂性， 人体临床试验所需的监管程序以及将研究产品用于 患者培训的一个新颖且非常成功的方面是在沟通、冲突和 解决问题的能力，人际关系和小组互动的能力。这些技能对于成功地在 跨学科的团队，并帮助在成功和保留少数民族学生的代表。 总的来说，这些机会将为学生在学术界内外的职业生涯做好准备， 推进NIH在卫生和人类服务方面加强生物医学研究的目标， 整体因此，我们实施的计划填补了翻译应用中的一个重大培训空白， 传统神经生物学、干细胞或临床神经系统疾病通常不符合干细胞生物学 研究生课程。

PROJECT SUMMARY Stem cell medicine promises to revolutionize the treatment of human diseases and injuries, and has captured the hopes of the scientific community and the public alike. Perhaps nowhere is the potential of stem cells to treat human disease and injury more promising than for neurologic disorders. Traveling a path from “bench to bedside” is still a relatively new opportunity for researchers and provides novel challenges for training graduate students. Historically, pre-doctoral neuroscience students were trained in understanding the basic biological mechanisms underlying how the brain functions and guides behavior, learning and memory, movement, and to identify the processes that become dysregulated and result in neurological disease. Indeed, the biological mechanisms and causes for many neurological diseases have been identified, paving the way for the generation of transgenic animals and model systems to study neurological disorders. Critically, these advances, combined with the advent of stem cell biology, open a path to treat neurologic diseases with transplantation of stem cell derived cell populations or to develop treatment strategies in stem-cell derived cell models. This is a path that requires training beyond basic science. Accordingly, the goal of our Training Program in Stem Cell Translational Medicine for Neurological Disorders is to provide opportunities for pre-doctoral trainees to perform translational "bench" research that could treat human neurological disease and injury, in the spirit of the NIH vision for translational medicine. Additionally however, to meet the challenges presented by the advent of this new frontier, we provide opportunities for trainees in the clinical aspects of the disease they are studying. These opportunities enable identification of therapeutic targets with the greatest relevance to human disease, inform preclinical safety and efficacy testing, and define preclinical outcome measures that parallel clinical metrics. Furthermore, this program familiarizes trainees with the complexities of the regulatory processes required for human clinical trials and the business of taking research products to patients. A novel, and highly successful, aspect of the training is coaching in communication, conflict resolution, interpersonal and group interactions skills. These skills are important both for success in working in interdisciplinary teams and for helping in the success and retention of underrepresented minority students. Taken together, these opportunities will prepare students for careers inside and outside academia and will advance the NIH goals of enhancing biomedical research in the context of health and human services as a whole. The program we have implemented thus fills a significant training gap in the translational application of stem cell biology to neurological disorders, not typically met by traditional neurobiology, stem cell or clinical graduate programs.