Neuro Therapeutics Research Institute (1 of 6)

研究联盟的主要目标是实施一个高度集成的、 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 以脆性X-相关性震颤/共济失调综合征为主要表现的迟发性神经退行性疾病 研究范式。FXTAS是最常见的单基因疾病之一，会导致震颤、共济失调和 痴呆症。该联盟解决的根本挑战是如何开发和集成 实现(和测量)对中枢神经系统紊乱的有针对性的治疗反应所需的各种成分。 该联盟将通过形成一个高度整合的Four Research研究核心来应对这一挑战 这些项目总共涉及30多名来自化学等不同学科的研究人员， 细胞神经生理学，发育儿科学，老鼠行为，神经成像，神经学，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)和认知神经科学 (项目4)，都有制定治疗干预措施和量化手段的共同目标 用来评估它们的疗效。T90培训部分将增强 通过进一步将不同项目的研究工作相互交织在一起，建立了一个合作伙伴关系。主体(非传统) T90的目标是加强联盟的主要研究目标。 该联盟将被组织为一个研究所(神经治疗研究所；NTRI)，有一个 财团指导委员会(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，并听取社会各界的意见。该财团包括 六个组成部分、一个行政单位(U54)、四个研究项目(R01)和一个博士后培训 组件(T90)。U54构成部分将促进整个联合体的目标和目的，并 提供智慧领导力，以最大限度地提高所有组件的集成度。这将导致 联盟可以作为一个强大的、敬业的教职调查人员社区蓬勃发展的环境， 受训人员和对神经退行性疾病领域做出重大贡献的工作人员 治疗这些疾病的疗法的发展。财团结构要求具有重要意义 组织规划和认真的管理和监督，以实现其科学性和教育性 目标。这一U54构成部分的职责包括一般行政、预算监督、协调 联合体活动(会议、座谈会和会议)、培训和外联管理、数据 分享和保护人类主体和动物。

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.