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.

资助父母奖的项目摘要（总体） 阿尔茨海默氏病（AD）是痴呆症，残疾和死亡的主要原因。尽管最近 我们对广告潜在基本生物学机制的理解的进步，我们尚不知道如何防止 AD或具有批准的疾病修改干预措施。两者都必须减慢或阻止痴呆症的生长 流行率。 《国家阿尔茨海默氏症计划法》（NAPA）试图在2025年预防和有效治疗广告 通过有关病因，早期检测和治疗的创新研究。为了支持纳帕的目标， NIA赞助的2015年阿尔茨海默氏病研究峰会上确定的研究领域是 下一代AD动物模型的发展将在临床前证明具有更大的预测性 研究并加速了药物测试管道。虽然我们目前的广告动物模型已提供 对AD疾病机制的多种新颖见解，到目前为止，尚未成功地用于预测 已进入AD临床试验的疗法的有效性。印第安纳大学（IU）/杰克逊 实验室（JAX）阿尔茨海默氏病精确模型中心（IU/JAX ADPMC）将利用IU的 神经退行性研究的优势，包括25年作为NIA支持的阿尔茨海默氏病 Jax的八十年专业知识中的中心（ADC）和临床前药物测试方面的大量专业知识 在哺乳动物遗传学和疾病建模中，以发展，验证和传播新的精密动物 阿尔茨海默氏病模型（AD）。此外，IU/JAX ADMPC还包含Sage Bionetworks提供 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和神经影像学，药品和世界领导者的临床专家 疾病精确动物模型的发展 - 具有集体能力的预测能力 疾病建模在国际舞台上出现时需求。这将允许IU/JAX ADPMC 有效高效地为广告科学界服务。 IU/JAX ADPMC将生成新广告 建模过程和管道，数据资源，研究结果和模型将迅速共享 通过Jax和Sage经过验证的传播管道，并通过NIA支持的广告中心， 全球学术医疗中心，研究机构和制药行业。最终，这个 将加速在动物模型中的进步，以获得最大的医疗利益。 IU/JAX ADPMC的具体目的是： 1。最大化人类数据集，以识别AD的推定变体，基因和生物标志物。 2。生成并表征下一代AD的鼠标模型。 3。验证AD的下一代小鼠模型并开发临床前测试管道。