Training Program in Stem Cell Translational Medicine for Neurological Disorders

项目总结 干细胞医学有望给人类疾病和创伤的治疗带来革命性的变化，并已获得 科学界和公众的希望都是如此。也许干细胞在任何地方都没有潜力 治疗人类疾病和伤害比治疗神经疾病更有前途。走过一条从“板凳”到 床边“对研究人员来说仍然是一个相对较新的机会，并为培养研究生提出了新的挑战 学生们。从历史上看，神经科学预科博士生接受的是理解基本生物学知识的培训。 大脑如何运作和指导行为、学习和记忆、运动以及 找出失控并导致神经系统疾病的过程。事实上，生物界 许多神经系统疾病的机制和病因已被确定，为 研究神经系统疾病的转基因动物和模型系统的产生。关键的是，这些 进步，结合干细胞生物学的出现，开辟了一条治疗神经系统疾病的途径 干细胞来源群体的移植或开发干细胞来源细胞的治疗策略 模特们。这是一条需要在基础科学之外进行培训的道路。因此，我们培训的目标是 神经疾病干细胞转化医学项目旨在为 博士前实习生将进行可治疗人类神经疾病的翻译性“板凳”研究 和损伤，在NIH对转化医学的愿景的精神中。然而，为了迎接挑战， 随着这一新领域的到来，我们为学员提供了临床方面的机会。 他们正在研究的疾病。这些机会使识别治疗靶点具有最大的 与人类疾病的相关性，告知临床前安全性和有效性测试，并定义临床前结果 衡量与临床指标平行的指标。此外，该计划使受训人员熟悉 人体临床试验所需的监管程序以及将研究产品带到 病人。培训的一个新的、非常成功的方面是在沟通、冲突方面的指导 解决问题、人际关系和团队互动的能力。这些技能对于在以下领域成功工作都很重要 在这方面，我要感谢各学科之间的合作，并帮助代表不足的少数族裔学生取得成功和留住他们。 总而言之，这些机会将为学生在学术界内外的职业生涯做好准备，并将 推进美国国立卫生研究院在卫生和公共服务方面加强生物医学研究的目标 完整的。因此，我们实施的计划填补了在翻译应用方面的一个重大培训空白 干细胞生物学到神经疾病，这是传统神经生物学、干细胞或临床所不能满足的 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.