BIOREPOSITORY OF HUMAN INDUCED PLURIPOTENT STEM CELLSFORCARDIOVASCULAR DISEASES (BHIPSC-CVD)- TASK A. CORE STUDY OPERATIONS

人类诱导多能干细胞用于心血管疾病 (BHIPSC-CVD) 的生物储存库 - 任务 A. 核心研究操作

• 批准号: 10721066
• 负责人: JOSEPH WU
• 金额: $ 15.73万
• 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/10721066
• 关键词: Address; Adopted; Adoption; African American population; 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.

该提案是为了响应 NIH/NHLBI 建立“心血管疾病人类诱导多能干细胞生物储存库”（以下简称“生物储存库”）的要求而制定的。拟建的斯坦福心血管研究所 (SCVI) 生物资源库将管理和管理更多的人类诱导多能干细胞 (iPSC) 系，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碍了个别研究实验室在其调查中采用 iPSC 模型，特别是在需要多种细胞系的研究中。为了解决这一瓶颈，拟建的生物储存库将维持我们现有的 iPSC 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) 捐献者（男女和不同种族/民族背景（非裔美国人、亚洲人、西班牙裔和白人））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获取未识别的表征数据。 在过去五年中，我们的团队已向心血管研究界的 250 多名研究人员提供了 2,500 多瓶 iPSC 和 iPSC 衍生心肌细胞 (iPSC-CM)。拟议的 SCVI 生物样本库将继承现有 SCVI iPSC 生物库的现有组织资产和设施，该生物库成立于 2014 年，通过 R24 机制（R24 HL117756；2014 年 4 月 15 日 - 2020 年 3 月 31 日；免费延期）在 NHLBI 的 5 年支持下建立。 SCVI 生物样本库位于斯坦福大学校园内，拥有 2,500 平方英尺的现代化设施，完全致力于拟议的生物样本库服务，并与学术研究活动隔离开来。斯坦福大学提供支持性的机构环境和世界一流的基础设施，包括 REDCap（研究电子数据捕获）和 STRIDE（斯坦福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以及用于资源管理的安全内部服务器。 我们组建了一支由专家研究人员和熟练员工组成的团队，他们拥有来自 SCVI 生物库的适当经验和培训。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，以满足生物样本库的运营和科学需求。将特别关注资源管理，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并管理材料转移、样品运输和物流。在拟议的 SCVI 生物库中，我们将与 NIH 合作，通过编写操作手册 (MOP) 和创建自动可跟踪请求系统来进一步整理我们的专业知识，这将使我们能够应对扩大 iPSC 交付的持续挑战，以促进未来的进步。