IU/JAX/Pitt MODEL-AD: MAPT-GR

IU/JAX/皮特 模型-AD：MAPT-GR

• 批准号: 10198518
• 负责人: Bruce T Lamb
• 金额: $ 41.09万
• 依托单位: INDIANA UNIV-PURDUE UNIV AT INDIANAPOLIS
• 依托单位国家: 美国
• 财政年份: 2016
• 资助国家: 美国
• 起止时间: 2016-09-30 至 2021-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10198518
• 关键词: Academic Medical Centers; Alzheimer disease prevention; Alzheimer' s Disease; Alzheimer' s disease model; Alzheimer’s disease biomarker; Animal Disease Models; Animal Model; Area; Award; Biological; Cause of Death; Cessation of life; Clinical; Clinical Trials; Communities; Data; Data Set; Dementia; Development; Disease; Disease model; Drug Industry; Early Diagnosis; Effectiveness; Elderly; Etiology; Funding; Genes; Genetic Models; Goals; Growth; Human; Indiana; Infrastructure; Institution; International; Intervention; Laboratories; Mammalian Genetics; Medical; Modeling; Nerve Degeneration; Parents; Preclinical Testing; Prevalence; Process; Research; Testing; Therapeutic; United States; United States National Institutes of Health; Universities; Variant; data resource; disability; drug testing; genetic technology; human disease; innovation; insight; mouse model; multidisciplinary; neuroimaging; next generation; novel; novel therapeutics; pre-clinical; preclinical study; prevent; symposium

PROJECT SUMMARY FOR FUNDED PARENT AWARD (OVERALL) Alzheimer's disease (AD) is a major cause of dementia, disability and death in the elderly. Despite recent advances in our understanding of basic biological mechanisms underlying AD, we do not yet know how to prevent AD or have an approved disease modifying intervention. Both are essential to slow or stop the growth in dementia prevalence. The National Alzheimer's Project Act (NAPA) seeks to prevent and effectively treat AD by 2025 through innovative research on etiology, early detection, and therapeutics. In support of NAPA's goals, one of the targeted areas of research identified at the NIA sponsored 2015 Alzheimer's Disease Research Summit was the development of the next generation of animal models of AD that will prove more predictive in preclinical studies and thus accelerate the drug testing pipeline. While our current animal models of AD have provided multiple novel insights into AD disease mechanisms, thus far they have not been successfully utilized to predict the effectiveness of therapies that have moved into AD clinical trials. The Indiana University (IU)/Jackson Laboratory (JAX) Alzheimer's Disease Precision Models Center (IU/JAX ADPMC) will leverage IU's strengths in neurodegenerative research including 25 years as an NIA-supported Alzheimer's Disease Center (ADC) and considerable expertise in preclinical drug testing with JAX's eight decades of expertise in mammalian genetics and disease modeling to develop, validate and disseminate new, precise animal models of Alzheimer's disease (AD). In addition, the IU/JAX ADMPC contains Sage Bionetworks to provide expertise in data organization and dissemination. The IU/JAX ADPMC brings together an international, multi-disciplinary team—including geneticists and genetics technology experts, quantitative and computational biologists, clinical experts in AD and neuroimaging, pharmacologists and world leaders in the development of precision animal models of disease—that possesses the collective ability to foresee disease modeling needs as they emerge on the international stage. This will allow the IU/JAX ADPMC to serve the AD scientific community effectively and efficiently. The IU/JAX ADPMC will generate new AD modeling processes and pipelines, data resources, research results and models that will be swiftly shared through JAX's and Sage's proven dissemination pipelines and through the NIA-supported AD Centers, academic medical centers, research institutions and the pharmaceutical industry worldwide. Ultimately, this will accelerate the application of advances in animal models for the greatest possible medical benefit. The Specific Aims of the IU/JAX ADPMC are: 1. Maximize Human Datasets to Identify Putative Variants, Genes and Biomarkers for AD. 2. Generate and Characterize the Next Generation of Mouse Models of AD. 3. Validate the Next Generation of Mouse Models of AD and Develop a Preclinical Testing Pipeline.

资助家长奖项目总结(总体) 阿尔茨海默病(AD)是导致老年人痴呆、残疾和死亡的主要原因。尽管最近 我们对阿尔茨海默病基本生物学机制的了解进展，我们还不知道如何预防 或有经批准的疾病修改干预措施。两者都是减缓或阻止痴呆症增长的关键。 流行率。《国家阿尔茨海默氏症项目法案》寻求在2025年前预防和有效治疗阿尔茨海默病 通过病因学、早期发现和治疗方面的创新研究。为了支持国家适应行动方案的目标，其中一个 在NIA主办的2015年阿尔茨海默病研究峰会上确定的目标研究领域是 下一代AD动物模型的发展将证明在临床前更具预测性 研究，从而加快药物测试管道。虽然我们目前的AD动物模型提供了 对AD疾病机制的多种新见解，到目前为止还没有成功地用于预测 已经进入AD临床试验的治疗方法的有效性。印第安纳大学(IU)/杰克逊 实验室(JAX)阿尔茨海默病精确模型中心(IU/JAX ADPMC)将利用IU的 神经退行性研究的优势，包括作为NIA支持的阿尔茨海默病的25年 中心(ADC)，并凭借JAX公司80年的专业知识在临床前药物测试方面拥有丰富的专业知识 在哺乳动物遗传学和疾病建模方面，开发、验证和传播新的、精确的动物 阿尔茨海默病(AD)模型。此外，IU/JAX ADMPC包含Sage Bionnetworks以提供 在数据组织和传播方面的专业知识。IU/JAX ADPMC汇集了一个国际、 多学科团队-包括遗传学家和遗传技术专家，定量和 计算生物学家、AD和神经成像方面的临床专家、药剂学家和世界领先的 疾病精确动物模型的发展--具有集体预见能力 随着疾病在国际舞台上的出现，疾病建模需要。这将允许Iu/JAX ADPMC 有效、高效地为AD科学界服务。Iu/JAX ADPMC将生成新的AD 将迅速共享的建模流程和管道、数据资源、研究结果和模型 通过JAX和Sage久经考验的传播管道以及NIA支持的AD中心， 世界各地的学术医学中心、研究机构和制药行业。归根结底，这 将加快应用先进的动物模型，以获得最大可能的医疗效益。这个 IU/JAX ADPMC的具体目标是： 1.最大化人类数据集，以确定AD的假定变异、基因和生物标记物。 2.建立和鉴定下一代AD小鼠模型。 3.验证下一代阿尔茨海默病小鼠模型，开发临床前测试流水线。