The Jackson Laboratory Center for Precision Genetics
杰克逊精密遗传学实验室中心
基本信息
- 批准号:10469581
- 负责人:
- 金额:$ 212.5万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 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:
- 关键词:Advanced DevelopmentAdvocateAllelesAnimal ModelAreaBioinformaticsBiologyBiomedical ResearchCellsCharacteristicsClinicalClinical TreatmentClinical TrialsCommunitiesComplementDNA Sequence AlterationDataData AnalysesData CollectionDevelopmentDiseaseDisease modelElementsFoundationsFundingGenerationsGeneticGenetic ModelsGenomicsGoalsInfrastructureLaboratoriesLaboratory miceLinkMaintenanceMammalian GeneticsMedical GeneticsModelingMolecularMusNewly Diagnosed DiseaseOutcomePatientsPharmacologyPhenotypePhysiologyPrecision therapeuticsPreclinical TestingProcessReproducibilityResearchResearch DesignResearch PersonnelResourcesServicesSystemThe Jackson LaboratoryTherapeuticTimeTranslationsUncertaintyValidationWorkadvanced diseaseanimal facilitybaseclinical applicationdata disseminationdata frameworkdata sharingdesigndisease phenotypedrug discoveryexpectationexperienceflexibilitygenetic resourcegenetic technologygenome editinghuman diseasehuman modelin vivoinnovationmodel developmentmouse geneticsmouse modelnext generationnoveloutreachpatient populationpre-clinicalprecision geneticspreclinical studypredictive modelingprogramsrepositorysuccesstherapeutic developmenttherapeutic evaluationtool
项目摘要
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.
项目总体概要
杰克逊实验室 (JAX) 精准遗传学中心 (JCPG) 将开发和传播新的、
精确的小鼠模型是为尽可能匹配疾病特征而定制的
受苦的病人。通过临床专家、患者、倡导者合作发起的提名流程
或基金会,JCPG 指导委员会将选择并优先考虑开发和临床前模型
数据收集。 JCPG 将利用 JAX 在生物信息学、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密小鼠模型的管道。这些鼠标型号
然后将进入最先进的动物设施进行维护和保护,因为它们已做好准备
分发给世界各地的生物医学和研究实体。
JCPG 的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
不同疾病领域的专家和许多临床合作者。这个团队不仅汇聚了广泛
不仅在一系列人类疾病领域拥有专业知识,而且在运营一系列疾病方面也拥有丰富的持续经验
大规模公共资源服务。该小组具有与临床专家有效合作的能力
识别并描述出现的疾病建模需求,然后创建新的小鼠模型来服务
需要小鼠模型来开发新的临床的不同利益相关者的不同需求
治疗。
JCPG 的活动旨在实现以下五个目标。 1. 组装和完善一个全面的、
可扩展且灵活的流程,用于生成和表征人类精密小鼠模型
疾病。 2. 建立数据分析和传播基础设施,促进下一代的发展
使用面向公众的交互式流程生成预测模型。 3. 加快发展
通过将患者数据与新的精准动物模型联系起来为临床前提供信息,从而提供新颖的精准疗法
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设施,可扩展并促进对所有 JCPG 资源的访问
受到公众和生物医学界的欢迎,同时通过现有的技术推广和传播新的小鼠模型
基础设施。 5. 通过将 JCPG 与其他 JAX 程序、资源和外部集成来提高效率
面向服务,从而建立可扩展的、财务上可持续的模型开发渠道。
项目成果
期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
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Cathleen M Lutz其他文献
Cathleen M Lutz的其他文献
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{{ truncateString('Cathleen M Lutz', 18)}}的其他基金
Project 4: Therapeutic Gene Editing for Rett Syndrome
项目 4:雷特综合征的治疗性基因编辑
- 批准号:
10668770 - 财政年份:2023
- 资助金额:
$ 212.5万 - 项目类别:
Interrogation of Neurological Pathologies Associated with Mutations in Kif1a
与 Kif1a 突变相关的神经病理学研究
- 批准号:
10728701 - 财政年份:2023
- 资助金额:
$ 212.5万 - 项目类别:
The Mutant Mouse Resource and Research Center at The Jackson Laboratory
杰克逊实验室突变小鼠资源和研究中心
- 批准号:
10400428 - 财政年份:2021
- 资助金额:
$ 212.5万 - 项目类别:
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