Clinical Translational Core (CTC)

临床翻译核心 摘要 我们IDDRC临床翻译核心(CTC)的目标是加速翻译 通过与Basic合作，研究神经发育障碍的新疗法 科学家和临床医生，以及培训未来的翻译神经科学的领导者。所提供的服务 支持翻译工作的临床翻译核心包括三个子核心：1)以患者为基础的临床前 由生物库和基于诱导多能干细胞(IPSC)的人神经元组成的服务 2)临床表型服务，由神经行为股和神经生理学股组成， 3)临床试验和监管服务，包括监管单位和研究参与者 注册表。反恐委员会提供的一整套服务使调查人员能够采取综合方法 神经发育障碍的治疗发现，从临床前研究中的患者样本到 自然病史和介入临床试验的生物标记物开发。次要核心1-以患者为基础 临床前服务-保持监管部门的批准，以收集可用于初诊的患者样本 研究或生成IPSC系和人类神经元单位内的神经元分化。子- 核心2-临床表型鉴定服务-提供专业知识和高密度脑电设备 作为直接表型通过神经心理评估和神经生理记录。次核心3- 临床试验和监管服务-提供监管框架方面的专家咨询支持 临床研究，使核心和IDDRC调查员能够为所有人提供服务 背景。促进基础科学研究人员和年轻人参与临床研究 调查人员，分核心协助准备法规文件，培训研究人员， 患者招募和数据分析。患者招募由研究参与者注册中心支持，a 收集了30,000多名愿意参与临床研究的个人，其中包括患有无数 神经发育诊断，他们的父母和兄弟姐妹，以及典型的发展中的对照。以确保 核心的努力集中在将为知识分子和 发育性残疾人口，核心与临床护理中心和患者密切相关 倡导团体，这两个团体都为该团体的活动和发展提供建议。通过这些努力， CORE在设计和推出临床前翻译和临床项目方面取得了公认的成功记录 对IDDRC的调查人员来说。

CLINICAL TRANSLATIONAL CORE ABSTRACT The objectives of the Clinical Translational Core (CTC) of our IDDRC are to accelerate the translation of research discoveries into new treatments for neurodevelopmental disorders, through collaboration with basic scientists and clinicians, as well as to train future leaders in translational neuroscience. Services offered by the Clinical Translational Core to support translational work include three sub-cores: 1) Patient-based Preclinical Services, comprised of a Biorepository and the induced pluripotent stem cell (iPSC) based Human Neuron Unit, 2) Clinical Phenotyping Services, consisting of the Neurobehavioral Unit and the Neurophysiology Unit, and 3) Clinical Trial and Regulatory Services, containing the Regulatory Unit and Research Participant Registry. The suite of services offered by the CTC enables investigators to pursue an integrated approach to therapeutic discovery for neurodevelopmental disorders, from patient derived samples in preclinical research to biomarker development to natural history and interventional clinical trials. Sub-core 1 - Patient Based Preclinical Services - maintains the regulatory approval to collect patient samples that can be used for primary research or for the generation of iPSC lines and neuronal differentiation within the Human Neuron Unit. Sub- core 2 - Clinical Phenotyping Services - provides expertise and access to high density EEG equipment as well as direct phenotyping through neuropsychological assessment and neurophysiological recording. Sub-core 3 - Clinical Trial and Regulatory Services - provides expert consultation in the regulatory framework supporting clinical research which enables both the services provided within the Core and IDDRC investigators of all backgrounds. To facilitate engagement in clinical research by basic science researchers and young investigators, the sub-core assists in the preparation of regulatory documents, the training of study staff, patient recruitment and data analysis. Patient recruitment is supported by the Research Participant Registry, a collection of over 30,000 individuals willing to participate in clinical research, including patients with a myriad of neurodevelopmental diagnoses, their parents and siblings, and typically developing controls. To ensure that the efforts of the Core are focused in areas that will produce meaningful benefits for the intellectual and developmental disability population, the Core is strongly connected to clinical care centers and patient advocacy groups, both of which advise the activity and development of the group. Through these efforts, the Core has a proven record of success in designing and launching preclinical translational and clinical projects for IDDRC investigators.