BIOREPOSITORY OF HUMAN INDUCED PLURIPOTENT STEM CELLSFORCARDIOVASCULAR DISEASES (BHIPSC-CVD) - TASK AREA B

用于心血管疾病的人源多能干细胞生物储存库 (BHIPSC-CVD) - 任务领域 B

• 批准号: 10721067
• 负责人: JOSEPH WU
• 金额: $ 83.35万
• 依托单位: STANFORD UNIVERSITY
• 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 2023-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10721067
• 关键词: Address; Adopted; Adoption; African American population; Area; Asian population; Cardiovascular Diseases; Cardiovascular system; Cell Line; Cells; Clinical; Clinical Informatics; Collection; Communities; Data; Data Management Resources; Databases; Development; Dilated Cardiomyopathy; Disease; Disease model; Drug Screening; Environment; Future; Hispanic Populations; Human; Hypertrophic Cardiomyopathy; Individual; Infrastructure; Inherited; Institutes; Laboratory Research; Leadership; Logistics; Manuals; Modernization; Molecular; Monoclonal Antibody R24; National Heart, Lung, and Blood Institute; Patients; Protocols documentation; Research; Research Activity; Research Personnel; Resources; Sampling; Secure; Services; Shipping; Ships; Speed; Standardization; System; Technical Expertise; Therapeutic Human Experimentation; Training; Translational Research; United States National Institutes of Health; Vial device; Work; biobank; cost; data repository; electronic data; experience; induced pluripotent stem cell; induced pluripotent stem cell derived cardiomyocytes; induced pluripotent stem cell technology; operation; prevent; racial and ethnic; racial diversity; recruit; response; scale up; sex; square foot; stem; stem cell delivery; stem cell model; web site

This proposal is prepared in response to the NIH/NHLBI request to establish a "Biorepository of Human Induced Pluripotent Stem Cells for Cardiovascular Diseases" (hereafter "Biorepository"). The proposed Stanford Cardiovascular Institute (SCVI) Biorepository will curate and manage an expanded collection of human induced pluripotent stem cell (iPSC) lines and provide investigators with iPSC materials generated using standardized protocols, with the overall objective of promoting the continued adoption and development of iPSC technology in cardiovascular research. Human iPSCs have revolutionized disease modeling, drug screening, and therapeutic research. However, it is also recognized that the need for specialized technical expertise and the associated cost have largely prevented individual research laboratories from adopting iPSC models in their inquiries, particularly in studies requiring multiple cell lines. To address this bottleneck, the proposed Biorepository will maintain our existing collection of iPSC lines and further expand the collection of iPSCs by recruiting 1,000 healthy, hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy (DCM) donors of both sexes and diverse racial/ethnic backgrounds (African Americans, Asians, Hispanics, and Whites). We will continue to provide cell materials to investigators at minimal shipping costs as well as open access to de- identified characterization data on our public website. In the past five years, our team has delivered over 2,500 vials of iPSCs and iPSC-derived cardiomyocytes (iPSC-CMs) to over 250 investigators in the cardiovascular research community. The proposed SCVI Biorepository will inherit established organizational assets and facilities of the present SCVI iPSC Biobank, which was established in 2014 under a 5-year NHLBI support through the R24 mechanism (R24 HL117756; 4/15/2014 - 3/31/2020; no-cost extension). The SCVI Biorepository is located on the Stanford campus and occupies 2,500 sq. ft of modern facilities that are wholly dedicated to the proposed biobanking service and sandboxed from academic research activities. Stanford offers a supportive institutional environment and world-class infrastructure, including the REDCap (Research Electronic Data Capture) and STRIDE (Stanford Translational Research Integrated Database Environment) clinical informatics data management systems, campus-wide high-speed secure networks, and secure internal servers for resource management. We have assembled a team of expert investigators and skilled staff with suitable experience and training from our SCVI Biobank. Our leadership team, internal scientific advisory board, and three core staff teams (Administrative, Technical, and Recruitment teams) stand ready to work with NIH/NHLBI and external advisors to tackle operational and scientific needs of the Biorepository. Particular focus will be given to resource management to maintain existing resources and anticipate future expansion. We possess ample experience to handle service requests and administer material transfer, sample shipment, and logistics. In the proposed SCVI Biorepository, we will work with NIH to further codify our expertise by Authoring a Manual of Operations (MOP) and creating an automated trackable request system, which will us meet ongoing challenges of scaling up iPSC delivery to promote future advances.

本提案是应NIH/NHLBI关于建立“用于心血管疾病的人类诱导多能干细胞生物储存库”（以下简称“生物储存库”）的要求而编写的。拟议中的斯坦福大学心血管研究所（SCVI）生物库将策划和管理人类诱导多能干细胞（iPSC）系的扩大收集，并为研究人员提供使用标准化方案生成的iPSC材料，总体目标是促进iPSC技术在心血管研究中的持续采用和发展。 人类iPSC彻底改变了疾病建模、药物筛选和治疗研究。然而，人们也认识到，对专业技术知识的需求和相关成本在很大程度上阻止了个别研究实验室在其调查中采用iPSC模型，特别是在需要多个细胞系的研究中。为了解决这一瓶颈，拟议的生物储存库将维持我们现有的iPSC细胞系收集，并通过招募1,000名健康的、肥厚型心肌病（HCM）、扩张型心肌病（DCM）的性别和不同种族/民族背景（非洲裔美国人、亚洲人、西班牙裔和白人）的供体来进一步扩大iPSC的收集。我们将继续以最低的运输成本向研究人员提供细胞材料，并在我们的公共网站上开放访问去识别的表征数据。 在过去的五年里，我们的团队已经向心血管研究领域的250多名研究人员提供了超过2,500瓶iPSC和iPSC衍生的心肌细胞（iPSC-CM）。拟议的SCVI生物储存库将继承现有SCVI iPSC生物库的既定组织资产和设施，该生物库于2014年通过R24机制（R24 HL 117756; 2014年4月15日至2020年3月31日;无成本延期）在5年NHLBI支持下成立。SCVI生物储存库位于斯坦福大学校园内，占地2,500平方米。完全致力于拟议的生物库服务和沙盒从学术研究活动的现代化设施英尺。斯坦福大学提供了一个支持性的机构环境和世界一流的基础设施，包括REDCap（研究电子数据采集）和STRIDE（斯坦福大学转化研究集成数据库环境）临床信息学数据管理系统，校园范围内的高速安全网络和安全的内部服务器资源管理。 我们已经组建了一个由专家调查员和熟练的工作人员组成的团队，他们具有适当的经验和来自我们SCVI生物库的培训。我们的领导团队，内部科学顾问委员会和三个核心员工团队（行政，技术和招聘团队）随时准备与NIH/NHLBI和外部顾问合作，以解决生物储存库的运营和科学需求。将特别注重资源管理，以维持现有资源并预测未来的扩展。我们拥有丰富的经验来处理服务请求和管理材料转移，样品运输和物流。在拟议的SCVI生物储存库中，我们将与NIH合作，通过编写操作手册（MOP）和创建自动化可跟踪请求系统来进一步编纂我们的专业知识，这将使我们能够应对扩大iPSC交付以促进未来发展的持续挑战。