ESTABLISHING A MUTANT MOUSE REGIONAL RESOURCE AT THE JACKSON LABORATORY: AIDS

在杰克逊实验室建立突变小鼠区域资源：艾滋病

• 批准号: 8356887
• 负责人: LEAH RAE B DONAHUE
• 金额: $ 26.69万
• 依托单位: JACKSON LABORATORY
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-01-01 至 2011-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8356887
• 关键词: Acquired Immunodeficiency Syndrome; Affect; Animal Model; Archives; Bioinformatics; Biology; Biomedical Research; Communities; Coupled; Derivation procedure; Disease; Disease model; ES Cell Line; Explosion; Funding; Gene Targeting; Genes; Genetic; Genetically Engineered Mouse; Grant; Hereditary Disease; Human; Human Genetics; Life; Mus; Mutagenesis; Mutant Strains Mice; National Center for Research Resources; Pathology; Pharmaceutical Preparations; Phenotype; Play; Pluripotent Stem Cells; Prevention strategy; Principal Investigator; Production; Quality Control; Research; Research Infrastructure; Resources; Role; Safety; Scientist; Services; Source; System; Techniques; Technology; Testing; The Jackson Laboratory; Therapeutic; Transgenic Organisms; United States National Institutes of Health; biological systems; cost; experience; genome sequencing; human disease; induced pluripotent stem cell; mouse model; mutant; pre-clinical research; programs; public health relevance; repository; stem cell technology; virtual

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. Primary support for the subproject and the subproject's principal investigator may have been provided by other sources, including other NIH sources. The Total Cost listed for the subproject likely represents the estimated amount of Center infrastructure utilized by the subproject, not direct funding provided by the NCRR grant to the subproject or subproject staff. DESCRIPTION (provided by applicant): AIDS related. This application proposes to establish a fourth Mutant Mouse Regional Resource Center (MMRRC) repository at The Jackson Laboratory (JAX). For over 60 years mutant mice, with phenotypes mimicking human diseases, have played a critical role in understanding the genetics underlying mammalian biology and human genetic disorders. The advent of transgenic and gene targeting technologies, coupled with large scale mutagenesis and gene trap programs has led to a virtual explosion in the production and use of mutant and genetically engineered mice. These valuable resources can only be fully exploited if they are archived, managed, and made accessible to the scientific community by centralized repositories. The Jackson Laboratory's experience and capacity as a repository, the quality of its existing programs, its management systems, and its reputation for research excellence all demonstrate that JAX will be a strong addition to the MMRRC network. In this application, we propose to establish a JAX MMRRC that would: 1) acquire mutant strains; 2) import and archive mutant strains; 3) distribute strains as cryopreserved material, mice recovered from cryopreserved material, ES cell lines, or live mice; 4) implement a state-of-the-art quality control program; 5) provide customer support and technical service; 6) promote MMRRC strains; 7) have an interactive Repository management plan; 8) establish the bioinformatics infrastructure to operate and to interact with other MMRRC repositories, and 9) conduct research to add utility to JAX MMRRC strains by developing pluripotent stem cell resources from valuable Repository disease models using both standard derivation techniques and IPS technology. The JAX MMRRC will be fully functional upon funding and will offer experience and capacity to the existing MMRRC program. PUBLIC HEALTH RELEVANCE (provided by applicant): The JAX Mutant Mouse Regional Resource Center (MMRRC) repository proposed in this project will provide access to mouse models for human disorders and for basic biomedical research to understand biological systems affected by human diseases. With a sequenced genome and increasingly sophisticated stem cell technologies to manipulate it, the mouse remains the most powerful model organism for biomedical research. Genetically engineered mouse models with homologous human genes are critical for translational and pre-clinical research. They enable scientists to understand the causes and pathologies of human diseases so that prevention strategies or therapeutics for these diseases can be developed. In addition, therapies or drugs can be tested on mouse models prior to human trials to assess efficacy and safety.

该子项目是利用资源的众多研究子项目之一 由 NIH/NCRR 资助的中心拨款提供。子项目的主要支持 并且子项目的主要研究者可能是由其他来源提供的， 包括其他 NIH 来源。 子项目可能列出的总成本 代表子项目使用的中心基础设施的估计数量， NCRR 赠款不直接向子项目或子项目工作人员提供资金。 描述（由申请人提供）：与艾滋病相关。 该申请建议在杰克逊实验室 (JAX) 建立第四个突变小鼠区域资源中心 (MMRRC) 存储库。 60多年来，具有模仿人类疾病表型的突变小鼠在理解哺乳动物生物学和人类遗传疾病的遗传学方面发挥了关键作用。转基因和基因靶向技术的出现，加上大规模诱变和基因陷阱计划，导致突变小鼠和基因工程小鼠的生产和使用几乎呈爆炸式增长。这些宝贵的资源只有通过集中存储库进行归档、管理并供科学界访问，才能得到充分利用。杰克逊实验室作为存储库的经验和能力、现有项目的质量、管理系统以及卓越研究的声誉都表明 JAX 将成为 MMRRC 网络的有力补充。在此应用中，我们建议建立一个 JAX MMRRC，它将：1）获得突变菌株； 2）导入并存档突变株； 3)以冻存材料、从冻存材料中回收的小鼠、ES细胞系或活小鼠的形式分发菌株； 4) 实施最先进的质量控制程序； 5）提供客户支持和技术服务； 6) 促进MMRRC菌株； 7）有一个交互式的Repository管理计划； 8) 建立生物信息学基础设施来操作其他 MMRRC 存储库并与之交互，以及 9) 通过使用标准衍生技术和 IPS 技术从有价值的存储库疾病模型中开发多能干细胞资源，进行研究以增加 JAX MMRRC 菌株的实用性。 JAX MMRRC 将在资助后全面运作，并将为现有 MMRRC 计划提供经验和能力。 公共卫生相关性（由申请人提供）：本项目中提出的 JAX 突变小鼠区域资源中心 (MMRRC) 存储库将提供人类疾病小鼠模型和基础生物医学研究，以了解受人类疾病影响的生物系统。凭借基因组测序和日益复杂的干细胞技术来操纵它，小鼠仍然是生物医学研究中最强大的模型生物。具有同源人类基因的基因工程小鼠模型对于转化和临床前研究至关重要。它们使科学家能够了解人类疾病的原因和病理学，从而制定这些疾病的预防策略或治疗方法。此外，在人体试验之前，可以在小鼠模型上测试疗法或药物，以评估疗效和安全性。