The NINDS Human Cell and Data Repository

NINDS 人类细胞和数据存储库

• 批准号: 9150320
• 负责人: JAY Arnold TISCHFIELD
• 金额: $ 117.57万
• 依托单位: RUTGERS, THE STATE UNIV OF N.J.
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-09-30 至 2020-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9150320
• 关键词: Affect; Alzheimer' s Disease; Amyotrophic Lateral Sclerosis; Area; Automation; Biopsy; Blood; Blood Cells; Cataloging; Catalogs; Cell Line; Cells; Central Nervous System Diseases; Collaborations; Collecting Cell; Collection; Communities; DNA; Data; Databases; Derivation procedure; Development; Disease; Disease model; Dystonia; Emerging Technologies; Ensure; Ethics; Fibroblasts; Gene-Modified; Generations; Genomics; Goals; Housing; Human; Huntington Disease; Individual; Informatics; Instruction; International; Lymphocyte; Maintenance; Medicine; Methodology; Mission; Mutation; National Institute of Drug Abuse; National Institute of Mental Health; National Institute of Neurological Disorders and Stroke; Neurodegenerative Disorders; Neurons; Neurosciences; Online Systems; Paper; Parkinson Disease; Participant; Pharmaceutical Preparations; Play; Pluripotent Stem Cells; Positioning Attribute; Preclinical Drug Evaluation; Price; Process; Production; Protocols documentation; Publications; Quality Control; Recording of previous events; Regenerative Medicine; Reporting; Research; Research Personnel; Resources; Role; Sampling; Secure; Services; Shipping; Ships; Skin; Source; Specimen; Stem Cell Research; Stem cells; Technology; Testing; Therapeutic; Tissue Sample; Tissues; United States National Institutes of Health; Universities; Validation; Washington; Work; biobank; blastomere structure; cell registry; cell type; cost; design; exhibitions; falls; frontotemporal degeneration; genetic resource; genome editing; human disease; improved; induced pluripotent stem cell; interest; medical schools; meetings; nerve stem cell; news; novel; novel strategies; pluripotency; prospective; public health relevance; quality assurance; repaired; repository; research and development; sample collection; technology development; therapeutic development; tool; tool development; user-friendly; web portal; web site

 DESCRIPTION (provided by applicant): Human pluripotent stem cells that can give rise to almost any type of cell were originally isolated from embryonic cells and their utility as tools fo research and therapeutic development was severely restricted due to ethical considerations as well as the inability to derive stem cells from subjects with known disorders. The relatively recent development of technologies for deriving induced pluripotent stem cells (iPSCs) from cells that were previously thought to be "terminally" differentiated, such as skin or blood cells, has opened up nearly limitless new avenues for understanding human disease and developing novel approaches for treatment. By converting cells collected from affected individuals into iPSCs, and then to relevant cell types such as neurons for central nervous system disorders, we can create "disease models in a dish", cells that recapitulate the disorder and can be used to screen therapeutic drugs or study the manifestation of the disorder and elucidate its origins. The NINDS Repository was created to collect and distribute a variety of human cells such as skin fibroblasts and blood cells and convert these to iPSCs for research in the areas of neurodegenerative disease such as Parkinson's disease, Alzheimer's disease, Huntington's disease, amyotrophic lateral sclerosis, Dystonia and Frontotemporal Degeneration. Given the complexity and cost of generating iPSC lines of high quality, and the emerging importance of disease modeling and regenerative medicine, there is a tremendous need in the neuroscience field for a reliable centralized source of these materials. RUCDR Infinite Biologics (RUCDR) is uniquely positioned to provide this resource for the NINDS. RUCDR has a long and successful track record as a repository and genetic resource since its inception in 1998. RUCDR manages several major repositories for the NIH, including the NIMH Repository and Genomics Resource (NRGR) which encompasses the NIMH Stem Cell Resource. The mission of the RUCDR NINDS Stem Cell Repository will be to work with NINDS investigators and staff on all aspects of study management to collect, process and distribute the highest quality cell samples from participants in its studies. These will include the culturing of fibroblasts from skin biopsies, th isolation of lymphocytes from blood, and the derivation of iPSC lines from both cell types. RUCDR will then perform a comprehensive range of tests on the iPSCs to ensure their quality, pluripotency and identity. RUCDR will provide additional services to the global community, including the generation of neural stem cells (NSCs), different types of neurons, and genome editing to repair or introduce in iPSCs specific mutations that are implicated in human disease.

 描述（由申请人提供）：可以产生几乎任何类型细胞的人多能干细胞最初是从胚胎细胞中分离的，并且由于伦理考虑以及不能从患有已知疾病的受试者获得干细胞，它们作为研究和治疗开发工具的效用受到严重限制。从以前被认为是“终末”分化的细胞（如皮肤或血细胞）中获得诱导多能干细胞（iPSC）的技术的相对较新的发展为理解人类疾病和开发新的治疗方法开辟了几乎无限的新途径。通过将从受影响的个体收集的细胞转化为iPSC，然后转化为相关的细胞类型，如中枢神经系统疾病的神经元，我们可以创建“培养皿中的疾病模型”，这些细胞重现了疾病，可用于筛选治疗药物或研究疾病的表现并阐明其起源。 NINDS Repository的创建是为了收集和分配各种人类细胞，如皮肤成纤维细胞和血细胞，并将其转化为iPSC，用于神经退行性疾病领域的研究，如帕金森病，阿尔茨海默病，亨廷顿病，肌萎缩侧索硬化症，肌张力障碍和额颞叶变性。考虑到产生高质量iPSC系的复杂性和成本，以及疾病建模和再生医学的重要性，神经科学领域迫切需要这些材料的可靠集中来源。RUCDR Infinite Biologics（RUCDR）是唯一能够为NINDS提供这种资源的公司。自1998年成立以来，RUCDR作为一个储存库和遗传资源有着长期和成功的记录。RUCDR管理着NIH的几个主要知识库，包括NIMH知识库和基因组学资源（NRGR），其中包括NIMH干细胞资源。RUCDR NINDS干细胞储存库的使命是与NINDS研究人员和工作人员在研究管理的各个方面合作，收集，处理和分发研究参与者的最高质量细胞样本。这些将包括培养来自皮肤活检的成纤维细胞，从血液中分离淋巴细胞，以及从两种细胞类型中衍生iPSC系。RUCDR将对iPSC进行全面的测试，以确保其质量，多能性和身份。RUCDR将为全球社区提供额外的服务，包括神经干细胞（NSC），不同类型的神经元的生成，以及基因组编辑，以修复或引入与人类疾病有关的iPSC特定突变。