Training Program in Stem Cell Translational Medicine for Neurological Disorders

2013年，我们启动了神经系统疾病干细胞转化医学培训计划， （“培训计划”）在加州大学欧文分校（UCI）的前提下，这种培训是必不可少的 如果干细胞要成功地用于治疗神经系统疾病的话。九年后，我们相信， 随着越来越多的学术团体和生物技术公司探索这一潜力， 干细胞直接治疗或导致治疗人类疾病和损伤。翻译的三个主要领域 正在进行的研究：使用干细胞或其衍生物进行移植，以取代那些细胞 损伤或破坏，使用干细胞，包括患者来源的细胞，研究疾病过程 和机制，以及使用干细胞的分化产物来筛选可以缓解的药物 疾病或损害。近年来，CRISPR介导的基因编辑的加入和“脑”的发展， “类器官”为该领域研究人员的武器库增加了新的工具。干细胞的前景 治疗成为现实，不仅必须在这个快速发展的领域进行基础研究， 还必须通过临床前和临床开发将进展转化为临床实践。平移 方法必须直接传授。这次更新的目标是提供一个更好的环境来培养一个 新一代的科学家在干细胞生物学的翻译应用到神经科学。基础上 关键的成功和反馈，从学员在这个过程中，T32自成立以来，我们的更新 该计划将更好地服务于这一目的，包括（1）最先进的严格和可重复的湿实验室 科学采用训练的基本原则的严谨性和可重复性，以及定量和 统计方法;（2）临床经验，以了解和计划在临床背景下的研究工作 翻译;（3）关于将发现转移到临床试验和治疗的实践方面的知识 通过行业实习和苏和比尔格罗斯干细胞研究中心的资源;（4）沟通， （5）熟悉美国食品药品监督管理局（FDA） 临床试验的批准程序;以及（6）增加对促进多样性，公平性和 包含（DEI）。这些讲习班/培训内容受到普遍赞扬， 跨学科科学的成功至关重要，并提高所有学生的成功，特别是 由于对网络的重视和积极影响，代表性不足的学生。这六个要素 代表培训的独特方面，根据UCI的成功经验， 临床翻译

In 2013, we launched our Training Program in Stem Cell Translational Medicine for Neurological Disorders (“training program”) at the University of California, Irvine (UCI) with the premise that such training is essential if stem cells are to be used successfully to treat neurological disorders. Nine years later, we believe that this premise is even more true, as many more academic groups and biotechnology companies explore the potential of stem cells to directly treat, or lead to treatments for, human disease and injury. Three main area of translational research are being conducted: the use of stem cells or their derivatives for transplantation to replace those cells damaged or destroyed by injury, the use of stem cells, including patient derived cells, to study disease processes and mechanisms, and the use of differentiated products of stem cells to screen for drugs that could alleviate disease or damage. In recent years the addition of CRISPR-mediated gene editing and the development of “brain organoids” have added new tools to the arsenal of researchers in the field. For the promise of stem cell treatments to become a reality, not only must basic research advance in this rapidly evolving field, but these advances must also be translated through preclinical and clinical development into clinical practice. Translational approaches must be taught directly. The goal of this renewal is to provide an even better environment to train a new generation of scientists in the translational application of stem cell biology to neuroscience. Building upon key successes and feedback from the trainees over the course of this T32 since its inception, our updated program will even better serve this purpose and includes (1) state-of-the-art rigorous and reproducible wet lab science employing training in the fundamental principles of rigor and reproducibility, as well as quantitative and statistical methods; (2) clinical experiences to understand and plan research efforts in the context of clinical translation; (3) knowledge regarding practical aspects of moving discoveries to clinical trials and therapies through industry internships and Sue and Bill Gross Stem Cell Research Center resources; (4) communication, StrengthsFinder and personal interaction skills; (5) Familiarity with the Food and Drug Administration (FDA) approval processes for clinical trials; and (6) an increased commitment to promoting diversity, equity and inclusion (DEI). These workshops/elements of training, which were universally praised, represent skills that are critical for success in interdisciplinary science and enhance success for all students, particularly underrepresented students because of the focus on and positive impact of networking. These six elements represent unique aspects of training designed for this training program based on UCI’s experience of successful clinical translation.