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.

这个子项目是许多利用资源的研究子项目之一 由NIH/NCRR资助的中心拨款提供。子项目的主要支持 而子项目的主要调查员可能是由其他来源提供的， 包括其它NIH来源。 列出的子项目总成本可能 代表子项目使用的中心基础设施的估计数量， 而不是由NCRR赠款提供给子项目或子项目工作人员的直接资金。 描述（由申请人提供）：艾滋病相关。 本申请提出在杰克逊实验室（JAX）建立第四个突变小鼠区域资源中心（MMRRC）储存库。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基础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程序，导致突变和基因工程小鼠的生产和使用的虚拟爆炸。这些宝贵的资源只有通过集中储存库进行存档、管理并向科学界开放，才能得到充分利用。杰克逊实验室作为知识库的经验和能力、其现有项目的质量、管理系统及其卓越的研究声誉，都表明JAX将是MMRRC网络的一个强有力的补充。在本申请中，我们建议建立JAX MMRRC，其将：1）获得突变菌株; 2）进口和存档突变菌株; 3）将菌株作为冻存材料、从冻存材料中回收的小鼠、ES细胞系或活小鼠分发; 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）推广MMRRC菌株; 7）有一个交互式的知识库管理计划; 8）建立生物信息学基础设施，以操作和与其他MMRRC知识库交互，以及9）通过使用标准衍生技术和IPS技术从有价值的知识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 MMRC将在获得资金后全面运作，并将为现有的MMRC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）储存库将提供人类疾病和基础生物医学研究的小鼠模型，以了解受人类疾病影响的生物系统。随着基因组测序和日益复杂的干细胞技术来操纵它，老鼠仍然是生物医学研究中最强大的模式生物。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，以便制定这些疾病的预防策略或治疗方法。此外，治疗或药物可以在人体试验之前在小鼠模型上进行测试，以评估疗效和安全性。