Training in Translational Develomental Neuroscience

描述(申请人提供)：我们正在进入一个了解和治疗发育中的神经系统疾病的前所未有的机会的时代，这带来了巨大的痛苦和成本。遗传学、分子和细胞生物学、系统和行为神经科学以及生物信息学的最新进展为我们提供了强大的新工具和概念，既可以用来发现儿童神经疾病的原因，也可以用来创造有效的治疗方法。在减轻儿童神经功能障碍的负担方面，并不缺乏好的想法和工具来切实有效地工作，并取得成功。但成功也有重要的障碍：神经科学研究界对发育中的神经系统疾病的重视程度较低；工业界对发育障碍的重视程度较低；神经科学的发现与新疗法的提供之间存在脱节。为了推进成功解决发育障碍的议程，我们需要创建一支才华横溢的研究人员队伍，他们的培训将为他们在翻译发育神经科学领域发挥领导作用做好准备。我们提出了一项培训计划，将临床儿童神经学和基础或临床科学研究方面的卓越与理解新见解如何转化为护理进步所需的经验结合起来。该计划由专门的教师组织，由斯坦福大学医学院支持，并聘请了一批才华横溢的导师，包括：1)在临床儿童神经学方面的卓越表现，2)发展神经科学方面的指导基础或临床研究，3)临床试验设计和实施方法方面的经验，4)翻译研究方面的培训，以及5)接触用于定义和开发新疗法的概念和方法。“转化发育神经科学”项目将培养出能够成功地将发育神经科学的见解转化为新疗法的儿童神经学家。

DESCRIPTION (provided by applicant): We are entering an era of unprecedented opportunities for understanding and treating disorders of the developing nervous system, which exact immense suffering and cost. Recent advances in genetics, molecular and cellular biology, systems and behavioral neuroscience, and bioinformatics have provided us with powerful new tools and concepts that can be used to both discover the cause of childhood neurological disorders and to create effective therapies. There is no paucity of good ideas and tools with which to work tangibly and with success toward alleviating the burden of neurological disorders in children. But there are important roadblocks to success: the low priority given to disorders of the developing nervous system by the neuroscience research community; the low priority given to developmental disorders by industry; and the disconnection between neuroscience discovery and the delivery of new treatments. Moving forward an agenda that successfully addresses developmental disorders requires that we create a talented cadre of investigators whose training prepares them to take on leadership roles in translational developmental neuroscience. We propose a training program that combines excellence in clinical Child Neurology and in basic or clinical science research with the experience needed to understand how new insights are translated into advances in care. Organized by a dedicated faculty, supported by Stanford University School of Medicine, and engaging a talented group of mentors, the program incorporates: 1) excellence in clinical Child Neurology, 2) mentored basic or clinical research in developmental neuroscience, 3) experience in the methods of design and conduct of clinical trials, 4) training in translational research and 5) exposure to the concepts and methods used to define and develop new therapies. The program in "Translational Developmental Neuroscience" will produce child neurologist-neuroscientists that can successfully translate developmental neuroscience insights into new treatments.