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: 10497610
• 负责人: JOSEPH WU
• 金额: $ 82.05万
• 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 2022-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10497610
• 关键词: African American; Area; Asians; Blood specimen; Cardiac Myocytes; Cardiotoxicity; Cardiovascular Diseases; Cardiovascular system; Cell Line; Cells; Collection; Development; Dilated Cardiomyopathy; Disease; Disease model; Drug Screening; Drug toxicity; Exhibits; Genotype; Growth; Hispanics; Human; Hypertrophic Cardiomyopathy; In Vitro; Individual; Individual Differences; Institutes; Medical History; Minority; National Heart, Lung, and Blood Institute; Patients; Phenotype; Property; Protocols documentation; Research; Research Personnel; Resources; Services; Signal Transduction; Standardization; biobank; cell type; drug discovery; drug use screening; induced pluripotent stem cell; induced pluripotent stem cell technology; novel therapeutic intervention; novel therapeutics; patient response; racial and ethnic; racial diversity; recruit; regenerative therapy; self-renewal; stem; transcriptome; treatment response

Since their 2006 discovery, human induced pluripotent stem cells (iPSCs) are applied in three major areas: disease modeling, drug discovery, and regenerative therapy. The cells hold inherent properties to self-renew and with the appropriate growth and signaling factors, they can differentiate into any cell type, such as induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs) that retain the genotype of individuals from whom they were derived from and exhibit the phenotypic features of disease in vitro. The use of iPSCs and iPSC-CMs enable researchers to understand disease mechanisms, screen for drug toxicity, and predict patient responses to new drugs. To promote the availability and development of iPSC technology in cardiovascular research, the Stanford Cardiovascular Institute (SCVI) Biorepository of Human Induced Pluripotent Stem Cells for Cardiovascular Diseases (BhiPSC-CVD) curates and manages a collection of human iPSC lines, as well as provides investigators with generated iPSC materials using standardized protocols. The specific aims of BhiPSC-CVD are to: 1) recruit dilated cardiomyopathy, hypertrophic cardiomyopathy, and healthy patients from diverse racial and ethnic backgrounds with a particular emphasis on minorities (e.g., African Americans, Asians, Hispanics, and Whites); 2) create and maintain iPSC lines from the patient pool or investigator-provided de-identified blood samples; 3) perform cardiotoxicity drug screening using iPSCs and their derivatives (e.g., iPSC-CMs); 4) acquire patient medical history and transcriptome information; and 5) provide NHLBI investigators with the necessary iPSC expertise, services, and resources. Overall, BhiPSC-CVD enables research towards greater understanding of individual differences in pathobiology and in response to treatment, as well as developing and optimizing novel therapeutic strategies to treat and cure cardiovascular disease.

自2006年发现以来，人类诱导多能干细胞（iPSC）主要应用于三个领域：疾病建模，药物发现和再生治疗。 这些细胞具有自我更新的固有特性，并且在适当的生长和信号传导因子的作用下，它们可以分化成任何细胞类型，例如诱导多能干细胞衍生的心肌细胞（iPSC-CM），其保留了它们所来源的个体的基因型，并在体外表现出疾病的表型特征。 iPSC和iPSC-CM的使用使研究人员能够了解疾病机制，筛选药物毒性，并预测患者对新药的反应。 为了促进iPSC技术在心血管研究中的可用性和发展，斯坦福大学心血管研究所（SCVI）心血管疾病人类诱导多能干细胞生物储存库（BhiPSC-CVD）策划和管理一系列人类iPSC细胞系，并使用标准化方案为研究人员提供生成的iPSC材料。 BhiPSC-CVD的具体目标是：1）招募来自不同种族和民族背景的扩张型心肌病、肥厚型心肌病和健康患者，特别强调少数民族（例如，非裔美国人、亚洲人、西班牙裔和白人）; 2）从患者库或制造商提供的去识别血液样品中产生并维持iPSC系; 3）使用iPSC及其衍生物（例如，iPSC-CM）; 4）获取患者病史和转录组信息; 5）为NHLBI研究人员提供必要的iPSC专业知识，服务和资源。 总的来说，BhiPSC-CVD使研究能够更好地了解病理生物学和治疗反应的个体差异，以及开发和优化治疗和治愈心血管疾病的新型治疗策略。