Neuro Therapeutics Research Institute

研究联盟的主要目标是实施一个高度综合的， 跨学科的方法来开发神经遗传性疾病的靶向分子疗法，使用 迟发性神经退行性疾病脆性X相关震颤/共济失调综合征（FXTAS）是其主要的 研究范式FXTAS是最常见的单基因疾病之一，可导致震颤、共济失调和 痴呆联合会面临的根本挑战是如何发展和整合 实现（和测量）对CNS疾病的靶向治疗反应所需的各种成分。 该联盟将通过形成四个研究中心的高度集成的研究核心来应对这一挑战 这些项目总共涉及30多名来自化学、 细胞神经生理学，发育儿科学，小鼠行为学，神经影像学，神经病学，精神病学， 和认知神经科学项目跨越分子/细胞神经科学（项目1），动物 模型（项目2），定量表型和临床试验（项目3），认知神经科学 （项目4），所有这些项目的共同目标是制定治疗干预措施和量化手段 以评估其功效。T90训练部分将增强 通过进一步交织在项目的研究工作的联盟。非传统（Non-Traditional） T90的目标是加强联盟的主要研究目标。 该联盟将组织为一个研究所（神经治疗研究所; NTRI）， 联盟指导委员会（CSC）、内部咨询委员会（CIAC）和社区咨询委员会 提供科学和行政监督以及社区的投入。联合体包括： 六个组成部分，一个行政单位（U 54），四个研究项目（R 01），和博士后培训 组分（T90）。U 54组件将促进整个联合体的目标和目的， 提供知识领导，以最大限度地整合所有组件。这将导致 环境中，该联盟可以蓬勃发展，作为一个强大的，专门的社区教师调查， 学员和工作人员，使神经退行性疾病领域的重大贡献， 治疗这些疾病的疗法的发展。联合体结构要求 组织规划和认真的管理和监督，以实现其科学和教育 目标.这一U 54部门的职责包括一般行政管理、预算监督、 联合会活动（会议、座谈会和大会）、培训和外联管理、数据 分享和保护人类受试者和动物。

The principal objective of the ResearchConsortium is implementation of a highly integrated, interdisciplinary approach to develop targeted molecular therapeutics for neurogenetic disorders, using the late-onset neurodegenerative disorder fragile X-associatedtremor/ataxia syndrome (FXTAS)as its principal research paradigm. FXTAS is one of the most common single-gene disorders leading to tremor, ataxia, and dementia. The fundamental challenge addressed by the Consortium is how to develop and integrate the various components required to achieve (and measure) a targetedtherapeutic response to a CNSdisorder. The Consortium will meet this challenge by forming a highly-integrated research core of fourResearch Projects that, in aggregate, involve more than thirty investigators from disciplines as diverse as chemistry, cellular neurophysiology, developmental pediatrics, mouse behavior, neuroimaging, neurology, psychiatry, and cognitive neuroscience. Projects span domains of molecular/cellular neuroscience (Project 1), animal models (Project 2), quantitative phenotyping and clinical trials (Project3), and cognitive neuroscience (Project 4), all with the common objectives of developing therapeutic interventions and quantitative means for assessing their efficacy. A T90 training component will enhance the power and cohesivenessof the Consortium by further interweaving the research efforts across Projects. The principal (non-traditional) objective of the T90 is to enhance the primary research objectives of the Consortium. The Consortium will be organized as an Institute (NeuroTherapeutics Research Institute; NTRI),with a Consortium Steering Committee (CSC), Internal Advisory committee (CIAC), and Community Advisory Board to provide scientific and administrative oversight and input from the community. The Consortium comprises six components, an administrative unit (U54), four research projects (R01), and a postdoctoral training component (T90). The U54 Component will facilitate the goals and objectives of the entire Consortium and provide the intellectual leadership to maximize integration across all components. This will lead to an environment in which the Consortium can flourish as a strong, dedicated community of faculty investigators, trainees, and staff that makes a significant contribution to the field of the neurodegenerative diseases and to the development of therapeutics to treat these diseases. The Consortium structure requiressignificant organizational planning and careful administration and oversight to achieve its scientific and educational goals. The duties of this U54 component include general administration, budgetary oversight, coordination of Consortium activities (meetings, colloquia, and conferences),training and outreach management, data sharing, and protection of human subjects and animals.