Neuro Therapeutics Research Institute (1 of 6)

神经治疗研究所 (1 of 6)

基本信息

批准号：
7495693
负责人：
PAUL J HAGERMAN
金额：
$ 78.29万
依托单位：
UNIVERSITY OF CALIFORNIA AT DAVIS
依托单位国家：
美国
项目类别：
财政年份：
2007
资助国家：
美国
起止时间：
2007-09-15 至 2012-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/7495693
关键词：
Accounting Address Adult Advisory Committees Affect Age Alleles Alzheimer&apos s Disease Animal Model Animals Ataxia Autistic Disorder Behavior Behavioral Brain CGG repeat Cell model Cells Central Nervous System Diseases Chemistry Child Clinical Clinical Trials Cognitive Communities Complex Cross-Sectional Studies Cytoplasm Dementia Development Discipline Disease Education and Outreach Effectiveness Environment FMR1 FMR1 Gene FMRP Faculty Fragile X Syndrome Future Gene Targeting Genes Genetic Genotype Glutamates Goals Human Impaired cognition Individual Institutes Interdisciplinary Study Knock-in Mouse Lead Leadership Life Link Lithium Location Longevity Measures Medical Memantine Mentorship Messenger RNA Methodology Methods Modeling Molecular Molecular Target Mus Mutation Nature Nerve Degeneration Neurocognitive Neurodegenerative Disorders Neurology Neurons Neuroprotective Agents Neurosciences Nuclear Outcome Parkinson Disease Pathogenesis Pediatrics Pharmaceutical Preparations Phenotype Premature Ovarian Failure Principal Investigator Process Psychiatry RNA RNA I Reporting Research Research Infrastructure Research Institute Research Personnel Research Project Grants Risk Structure Syndrome Tetanus Helper Peptide Therapeutic Therapeutic Agents Therapeutic Human Experimentation Therapeutic Intervention Toxic effect Training Training Programs Transcript Translational Research Treatment Efficacy Treatment outcome Tremor Upper arm Variant base clinical phenotype cognitive neuroscience concept data management design early onset human study human subject improved interdisciplinary approach knock-down model development molecular domain mouse model neurogenetics neuroimaging neuropathology neurophysiology next generation post-doctoral training programs research study response symposium therapeutic target

Accounting Address Adult Advisory Committees Affect Age Alleles Alzheimer&apos s Disease Animal Model Animals Ataxia Autistic Disorder Behavior Behavioral Brain CGG repeat Cell model Cells Central Nervous System Diseases Chemistry Child Clinical Clinical Trials Cognitive Communities Complex Cross-Sectional Studies Cytoplasm Dementia Development Discipline Disease Education and Outreach Effectiveness Environment FMR1 FMR1 Gene FMRP Faculty Fragile X Syndrome Future Gene Targeting Genes Genetic Genotype Glutamates Goals Human Impaired cognition Individual Institutes Interdisciplinary Study Knock-in Mouse Lead Leadership Life Link Lithium Location Longevity Measures Medical Memantine Mentorship Messenger RNA Methodology Methods Modeling Molecular Molecular Target Mus Mutation Nature Nerve Degeneration Neurocognitive Neurodegenerative Disorders Neurology Neurons Neuroprotective Agents Neurosciences Nuclear Outcome Parkinson Disease Pathogenesis Pediatrics Pharmaceutical Preparations Phenotype Premature Ovarian Failure Principal Investigator Process Psychiatry RNA RNA I Reporting Research Research Infrastructure Research Institute Research Personnel Research Project Grants Risk Structure Syndrome Tetanus Helper Peptide Therapeutic Therapeutic Agents Therapeutic Human Experimentation Therapeutic Intervention Toxic effect Training Training Programs Transcript Translational Research Treatment Efficacy Treatment outcome Tremor Upper arm Variant base clinical phenotype cognitive neuroscience concept data management design early onset human study human subject improved interdisciplinary approach knock-down model development molecular domain mouse model neurogenetics neuroimaging neuropathology neurophysiology next generation post-doctoral training programs research study response symposium therapeutic target

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项目摘要

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.

ResearchContortium的主要目标是实施高度综合的，跨学科方法用于开发针对神经遗传疾病的靶向分子疗法，使用晚发神经退行性疾病脆弱的X相关tremor/共济失调综合征（FXTA）作为其主要研究范式。 FXTA是导致震颤，共济失调和的最常见的单基因疾病之一失智。财团提出的基本挑战是如何发展和整合实现（并测量）对CNSDISORDER的靶向性响应所需的各种组件。该财团将通过形成Fourresearch的高度集成研究核心来应对这一挑战总体而言，这些项目涉及来自化学等学科的三十多个研究人员，细胞神经生理学，发育小儿，小鼠行为，神经影像学，神经学，精神病学，精神病学，和认知神经科学。项目跨越分子/细胞神经科学的域（项目1），动物模型（项目2），定量表型和临床试验（Project3）和认知神经科学（项目4），所有这些都具有制定治疗干预措施和定量手段的共同目标用于评估其功效。 T90训练组件将增强联盟通过进一步交织在项目中的研究工作。校长（非传统） T90的目的是增强财团的主要研究目标。该财团将被组织为一家研究所（神经治疗研究所； NTRI），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的监督以及社区的意见。财团包括六个组件，一个行政部门（U54），四个研究项目（R01）和一个博士后培训组件（T90）。 U54组件将促进整个财团的目标和目标提供智力领导才能最大程度地提高所有组成部分的整合。这将导致财团可以作为一个强大，敬业的教师调查员社区蓬勃发展的环境，学员和员工为神经退行性疾病的领域做出了重大贡献治疗这些疾病的治疗剂的发展。财团结构需要专用组织计划以及仔细的管理和监督，以实现其科学和教育目标。该U54组件的职责包括总管理，预算监督，协调财团活动（会议，座谈会和会议），培训和外展管理，数据共享和保护人类受试者和动物。