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Clinical Translational Core (CTC)

临床转化核心（CTC）

基本信息

批准号：
10239465
负责人：
MUSTAFA SAHIN
金额：
$ 22.86万
依托单位：
BOSTON CHILDREN'S HOSPITAL
依托单位国家：
美国
项目类别：
财政年份：
2021
资助国家：
美国
起止时间：
2021-07-22 至 2026-05-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10239465
关键词：
Adaptive Behaviors Animal Behavior Area Basic Science Behavioral Biological Biological Assay Biological Markers Biology Biometry Blood Blood specimen Boston Cell Line Cells Child Care Child Psychiatry Clinic Clinical Clinical Research Clinical Trials Clinical Trials Design Cognitive Collaborations Collection Complex Consultations DNA Data Analyses Development Diagnosis Discipline Discrimination Disease Down Syndrome Drug Screening Electroencephalography Ensure Environment Equipment Event-Related Potentials Face Family Fibroblasts Fostering Fragile X Syndrome Future Generations Genetic Goals Growth Human Human Resources Image Improve Access Individual Infrastructure Institutional Policy Intellectual and Developmental Disabilities Research Centers Intellectual functioning disability Intervention Intervention Trial Language Mus Natural History Neurodevelopmental Disorder Neurofibromatoses Neuronal Differentiation Neurons Neurophysiology - biologic function Neuropsychological Tests Neuropsychology Neurosciences Parents Participant Patient Care Patient Recruitments Patients Pediatric Hospitals Pediatric Neurology Phenotype Physiological Physiology Population Positioning Attribute Pre-Clinical Model Preparation Process Registries Research Research Personnel Research Support Resources Rett Syndrome Sampling Scientist Services Siblings Technical Expertise Testing Therapeutic Tissues Training Translating Translational Research Tuberous sclerosis protein complex Work base bench to bedside biobank biomarker development biomarker performance cellular imaging clinical care clinical phenotype cohort community engagement density design genetic analysis high throughput screening human disease induced pluripotent stem cell mouse model multidisciplinary neurobehavioral neurophysiology next generation novel novel diagnostics novel therapeutic intervention patient advocacy group pre-clinical pre-clinical research programs protocol development rare genetic disorder screening social success tool translational neuroscience translational study treatment trial

项目摘要

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.

临床翻译核心摘要我们IDDRC的临床翻译核心（CTC）的目标是加速以下内容的翻译：研究发现神经发育障碍的新疗法，通过与基础科学家和临床医生，以及培养未来的领导者在转化神经科学。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）基于患者的临床前服务，包括生物储存库和基于人类神经元的诱导多能干细胞（iPSC） 2）临床表型分析服务，由神经行为科和神经生理科组成，和3）临床试验和监管服务，包括监管部门和研究参与者注册表反恐委员会提供的一系列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来源样本到生物标志物开发到自然史和干预性临床试验。子核心1 -基于患者临床前服务-保持监管批准，以采集可用于主要研究的患者样本研究或用于iPSC系的产生和人类神经元单元内的神经元分化。亚- 核心2 -临床表型分析服务-提供专业知识，并获得高密度EEG设备以及作为通过神经心理学评估和神经生理学记录的直接表型。次级核心3 - 临床试验和监管服务-提供监管框架方面的专家咨询，临床研究，使核心和IDDRC研究者提供的服务，背景促进基础科学研究人员和年轻人参与临床研究研究者，子核心协助编写监管文件，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登记处支持，收集了超过30，000名愿意参与临床研究的人，包括患有各种疾病的患者。神经发育诊断，他们的父母和兄弟姐妹，通常发展控制。确保核心的努力集中在将为知识和发展残疾人口，核心是密切联系的临床护理中心和病人倡导团体，这两个团体都为该团体的活动和发展提供咨询意见。通过这些努力， Core在设计和启动临床前转化和临床项目方面有着成功的记录 IDDRC调查员的