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.
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