NINDS Human Cell and Data Repository (U24)

NINDS 人类细胞和数据存储库 (U24)

• 批准号: 10226161
• 负责人: JENNIFER C MOORE
• 金额: $ 131.22万
• 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 2025-07-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10226161
• 关键词: Adult; Age; Alzheimer' s Disease; Amyotrophic Lateral Sclerosis; Automation; Biocompatible Materials; Biological; Biological databases; Biopsy; Blood; Blood Cells; Catalogs; Cell Line; Cell model; Cells; Central Nervous System Diseases; Certification; Clustered Regularly Interspaced Short Palindromic Repeats; Communities; Consultations; DNA; Data; Derivation procedure; Development; Disease; Disease Progression; Disease model; Documentation; Dystonia; Embryo; Ensure; Ethics; Exhibits; Family member; Fibroblasts; Foundations; Gene-Modified; Generations; Genetic; Genetic Research; Genetic Variation; Genotype; Goals; Human; Human Cell Line; Human Development; Human Genetics; Human Resources; Huntington Disease; Individual; Instruction; International; Internet; Logistics; Lymphocyte; Maintenance; Methodology; Mission; Modification; National Institute of Neurological Disorders and Stroke; Neurodegenerative Disorders; Neurons; Parkinson Disease; Pluripotent Stem Cells; Policies; Price; Privacy; Privatization; Process; Production; Protocols documentation; Quality Control; Reporter; Research; Research Personnel; Resources; Sampling; Secure; Services; Shipping; Ships; Site; Skin; Somatic Cell; Source; Stem Cell Research; System; Technology; Testing; Time; Tissue Sample; Toxicology; Trust; U-Series Cooperative Agreements; United States National Institutes of Health; Universities; Validation; Washington; Work; biobank; cell repository; cell type; clinical application; data management; data repository; design; drug discovery; drug use screening; exhibitions; flexibility; frontotemporal degeneration; genome editing; genome integrity; high standard; human disease; human subject; improved; induced pluripotent stem cell; innovation; interest; material transfer agreement; medical schools; meetings; nerve stem cell; nervous system disorder; news; online repository; operation; prospective; quality assurance; repository; research and development; sample collection; search engine; sex; stem cell genes; stem cells; therapeutic development; tool; user-friendly; volunteer; web app

NINDS Application Abstract Pluripotent stem cells were originally isolated from embryos but their utility as tools for 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. Human induced pluripotent stem cells (hiPSC) generated from adult somatic cells were first described in 2007. Since then, hiPSCs have been established from subjects with specific disorders (many with known genotypes) and then differentiated to relevant cell types such as neurons for central nervous system disorders. Using these hiPSC we can create “disease models in a dish” that can model cell development and disease progression and be used for drug screening and for toxicology testing. However, human genetic diversity has hampered the usefulness of individual hiPSC for disease modeling. Controls often consist of age and sex-matched non-affected family members or other subjects. The CRISPR (clustered regularly-interspaced short palindromic repeats)/Cas system can now generate isogenic hiPSC that will serve as better controls and help eliminate effects that are due to genetic background, thus providing a better understanding of mechanisms of human disease and development. The NINDS Human Cell and Data Repository (NHCDR) was established to collect, derive and distribute human cells such as skin fibroblasts and blood cells and convert these to iPSCs for research on neurodegenerative diseases such as Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, amyotrophic lateral sclerosis, Dystonia and Frontotemporal Degeneration. Since 1998, RUCDR Infinite Biologics® (RUCDR) has provided the NIH, private foundations and the global scientific community with the highest quality biomaterials, technical consultation, and logistical support for human genetics research. In 2011, RUCDR began providing its stem cell services to the NIH, including source cell and iPSC banking; iPSC generation; iPSC gene editing; and source cell and iPSC distribution. In 2015, RUCDR successfully competed for the NHCDR and in the past 5 years the catalog has grown to contain 268 FCL lines from 256 subjects and 259 iPSC lines from 132 subjects, including the addition of 7 isogenic pairs of iPSC. During this time RUCDR shipped 458 orders for 1538 cell lines to investigators around the world. The continuing mission of the NHCDR is to work with NINDS investigators and staff on all aspects of study management to collect, process and distribute the highest quality cell lines from human subjects. This effort includes the culturing of fibroblasts from skin biopsies, the isolation of lymphocytes from blood, and the derivation of iPSC lines from both cell types. RUCDR will continue to provide a comprehensive range of quality testing of iPSCs to ensure they conform to the highest standards. If requested, we will also provide additional services to the global community, including the generation of neural stem cells (NSCs) and different types of neurons. RUCDR will continue to exhibit scientific flexibility to grow and adapt to leading best practices in the stem cell field.

NINDS 申请摘要 多能干细胞最初是从胚胎中分离出来的，但它们可作为研究和治疗的工具 由于伦理考虑以及无法衍生干细胞，发育受到严重限制 来自患有已知疾病的受试者，由成人体细胞产生的人类诱导多能干细胞（hiPSC）。 细胞于 2007 年首次被描述。从那时起，hiPSC 就从具有特定特征的受试者中建立。 疾病（许多具有已知的基因型），然后分化为相关细胞类型，例如中枢神经元 使用这些 hiPSC，我们可以创建可以模拟细胞的“培养皿中的疾病模型”。 发育和疾病进展并可用于药物筛选和毒理学测试。 人类遗传多样性常常阻碍个体 hiPSC 在疾病建模中的实用性。 CRISPR（簇 规则间隔的短回文重复）/Cas 系统现在可以生成可用于服务的同基因 hiPSC 作为更好的控制并帮助消除遗传背景造成的影响，从而提供更好的 了解人类疾病和发育的机制。 NINDS 人类细胞和数据存储库 (NHCDR) 的建立是为了收集、导出和分发 人类细胞，如皮肤成纤维细胞和血细胞，并将它们转化为 iPSC 以进行研究 神经退行性疾病，如帕金森病、阿尔茨海默病、亨廷顿舞蹈病、 肌萎缩侧索硬化症、肌张力障碍和额颞叶变性 自 1998 年起，RUCDR Infinite。 Biologics® (RUCDR) 为 NIH、私人基金会和全球科学界提供了 2011年，为人类遗传学研究提供最优质的生物材料、技术咨询和后勤支持。 RUCDR 开始向 NIH 提供干细胞服务，包括源细胞和 iPSC 库； 2015年，RUCDR成功参赛。 对于 NHCDR，在过去 5 年中，该目录已发展到包含来自 256 个受试者的 268 个 FCL 系， 来自 132 名受试者的 259 个 iPSC 系，包括在此期间添加的 7 个同基因 iPSC 对。 向世界各地的研究人员运送了 458 份 1538 个细胞系的订单 NHCDR 的持续使命。 与 NINDS 研究者和工作人员在研究管理的各个方面合作，收集、处理和 分发来自人类受试者的最高质量的细胞系。这项工作包括培养来自人类的成纤维细胞。 皮肤活检、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 将继续提供 iPSC 的全面质量测试，以确保它们符合 如果有要求，我们还将向国际社会提供额外的服务，包括 神经干细胞（NSC）和不同类型神经元的产生将继续表现出来。 科学的灵活性，以发展和适应干细胞领域领先的最佳实践。