The Jackson Laboratory Center for Precision Genetics

杰克逊精密遗传学实验室中心

• 批准号: 10469581
• 负责人: Cathleen M Lutz
• 金额: $ 212.5万
• 依托单位: JACKSON LABORATORY
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-09-01 至 2025-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10469581
• 关键词: Advanced Development; Advocate; Alleles; Animal Model; Area; Bioinformatics; Biology; Biomedical Research; Cells; Characteristics; Clinical; Clinical Treatment; Clinical Trials; Communities; Complement; DNA Sequence Alteration; Data; Data Analyses; Data Collection; Development; Disease; Disease model; Elements; Foundations; Funding; Generations; Genetic; Genetic Models; Genomics; Goals; Infrastructure; Laboratories; Laboratory mice; Link; Maintenance; Mammalian Genetics; Medical Genetics; Modeling; Molecular; Mus; Newly Diagnosed Disease; Outcome; Patients; Pharmacology; Phenotype; Physiology; Precision therapeutics; Preclinical Testing; Process; Reproducibility; Research; Research Design; Research Personnel; Resources; Services; System; The Jackson Laboratory; Therapeutic; Time; Translations; Uncertainty; Validation; Work; advanced disease; animal facility; base; clinical application; data dissemination; data framework; data sharing; design; disease phenotype; drug discovery; expectation; experience; flexibility; genetic resource; genetic technology; genome editing; human disease; human model; in vivo; innovation; model development; mouse genetics; mouse model; next generation; novel; outreach; patient population; pre-clinical; precision genetics; preclinical study; predictive modeling; programs; repository; success; therapeutic development; therapeutic evaluation; tool

PROJECT SUMMARY OVERALL The Jackson Laboratory (JAX) Center for Precision Genetics (JCPG) will develop and disseminate new, precise mouse models that are tailored to match, as closely as possible, the disease characteristics of the afflicted patients. Through a nomination process initiated by collaborating clinical experts, patients, advocates or foundations, the JCPG Steering Committee will select and prioritize models for development and preclinical data collection. The JCPG will leverage JAX's long-established expertise in bioinformatics, mammalian genetics and disease modeling to integrate and refine existing resources, services and infrastructure to create a pipeline for development of precision mouse models for the biomedical community. These mouse models will then enter state-of-the-art animal facilities for maintenance and protection as they are prepared for distribution to biomedical and research entities worldwide. The JCPG expertise includes computational biologists, geneticists, molecular biologists, genetics technology experts and many clinical collaborators in diverse disease areas. This team not only brings together broad expertise in a range of human disease areas but also extensive ongoing experience in operating an array of large-scale public resource services. This group has a proven ability to work effectively with clinical experts to identify and delineate disease modeling needs as they emerge and then create new mouse models that serve the varied needs of the diverse set of stakeholders that need mouse models to develop new clinical treatments. The activities of the JCPG seek to achieve the following five goals. 1. Assemble and refine a comprehensive, scalable, and flexible pipeline for the generation and characterization of precision mouse models of human disease. 2. Build a data analysis and dissemination infrastructure that facilitates development of next- generation predictive models using an interactive public-facing process. 3. Accelerate the development of novel, precision therapeutics by linking patient data with new precision animal models to inform preclinical study design and generate the data needed for clinical trials. 4. Develop and deploy a robust outreach, project nomination, and project management infrastructure that expands and facilitates access to all JCPG resources by the public and biomedical community while promoting and distributing new mouse models through existing infrastructure. 5. Gain efficiencies by integrating the JCPG with other JAX programs, resources, and external- facing services and thereby establish a scalable, financially sustainable model development pipeline.

总体项目摘要 杰克逊实验室（JAX）精密遗传学中心（JCPG）将开发并传播新的， 精确的小鼠模型量身定制，以尽可能匹配，以匹配该疾病的特征 苦难的患者。通过合作临床专家发起的提名过程，患者，拥护者 或基金会，JCPG指导委员会将选择并优先考虑开发模型和临床前的模型 数据收集。 JCPG将利用Jax在生物信息学方面的悠久专业知识，哺乳动物 遗传学和疾病建模以整合和完善现有资源，服务和基础设施以创建 为生物医学界开发精确小鼠模型的管道。这些鼠标模型 然后，将在为维护和保护的情况下进入最先进的动物设施 分配给全球生物医学和研究实体。 JCPG专业知识包括计算生物学家，遗传学家，分子生物学家，遗传学技术 专家和许多不同疾病领域的临床合作者。这个团队不仅融合了 在一系列人类疾病领域的专业知识，但在运营一系列阵列方面也有丰富的持续经验 大规模公共资源服务。该小组具有与临床专家有效合作的可靠能力 识别并描述疾病建模的需求，然后创建新的鼠标模型 需要鼠标模型开发新临床的各种利益相关者的各种需求 治疗。 JCPG的活动试图实现以下五个目标。 1。组装和完整的， 可扩展和灵活的管道，用于生成和表征人类的精确小鼠模型 疾病。 2。建立数据分析和传播基础架构，以促进下一步的发展 使用互动公共的过程的生成预测模型。 3。加速发展 通过将患者数据与新的精密动物模型联系起来的新颖，精确治疗，以告知临床前 研究设计并生成临床试验所需的数据。 4。开发和部署强大的外展项目 提名和项目管理基础架构，可扩展和促进访问所有JCPG资源 公共和生物医学界的同时，通过现有 基础设施。 5。通过将JCPG与其他JAX程序，资源和外部 - 面对服务，从而建立可扩展的，财务可持续的模型开发管道。