Pulmonary Hypertension Breakthrough Initiative

肺动脉高压突破计划

• 批准号: 8756641
• 负责人: Serpil C. Erzurum
• 金额: $ 255.8万
• 依托单位: UNIVERSITY OF COLORADO DENVER
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-09-15 至 2018-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8756641
• 关键词: Abbreviations; Alabama; American; Bioinformatics; Blood; Blood Pressure; Blood Vessels; Blood specimen; Cardiac; Cardiovascular system; Cataloging; Catalogs; Categories; Cell Line; Cell Separation; Cells; Clinic; Clinical; Clinical Data; Colorado; Consult; Consultations; DNA analysis; Data; Data Analyses; Data Coordinating Center; Development; Diagnosis; Diagnostic; Diagnostic tests; Disease; Education; Enrollment; Funding; Future; Gene Expression; Genetic; Genomics; Goals; Heart; Human; Incidence; Informatics; Inherited; Institutes; Institutional Review Boards; Investigation; Lung; Lung Transplantation; Lung diseases; Medical Education; Medical Research; Michigan; Monoclonal Antibody R24; National Heart, Lung, and Blood Institute; Pathologic; Pathology; Patients; Pennsylvania; Pharmaceutical Preparations; Phenotype; Physiological; Predisposition; Process; Pulmonary Hypertension; Pulmonary artery structure; RNA; Recording of previous events; Research; Research Design; Research Infrastructure; Research Personnel; Research Support; Resources; Risk Factors; Role; Site; Specimen; Time; Tissues; Translations; Transplant Recipients; Transplantation; Universities; Woman; Work; World Health Organization; abstracting; base; biobank; blood pump; clinical care; database of Genotypes and Phenotypes; effective therapy; electronic data; flexibility; genome wide association study; heart cell; high throughput analysis; human tissue; hypertension control; material transfer agreement; meetings; member; mortality; next generation sequencing; novel; novel diagnostics; public health relevance; pulmonary arterial hypertension; success

DESCRIPTION (provided by applicant): Pulmonary Arterial Hypertension (PAH) is a heterogeneous group of diseases with high mortality, difficult diagnosis, limited available treatment, and no cure. The Pulmonary Hypertension Breakthrough Initiative (PHBI) is dedicated to comprehensive biobanking of human lungs with PAH and controls, supported by a detailed clinical annotation of the accrued specimens. Having started in 2006, the PHBI successfully developed a novel and unique infrastructure, whose success relied on the active participation of a highly integrated network of university-based sites with extensive expertise in each of the spheres of competency: excellence in clinical care of PAH (including patient accruals), lung transplantation, pathology, genetics, genomics, and cell isolation. The goal of this application is to leverage these unparalled resources and unique expertise into an R24-supported initiative to direct the bank to accrue specimens of disease-specific groups, which will be highly integrated with pathologic, genetic, and genomic subphenotypes pertaining to not only lung and blood specimens, but now expanding to the failing PAH heart. This proposal will be highly synergistic with the presently supported initiatives by the NHLBI in the field. The flexibilty of our infrastructure and governance will allow us to be decidedly responsive and integrated with any future NHLBI initiatives. We propose that this unique infrastructure will serve the pressing roles of advancing the translation of key discoveries to the patients with PAH and to aid in new discoveries that will have a long lasting impact in the field. The following specific aims will be pursued: Specific Aims of the Proposal 1) To establish a comprehensive biorepository of the specimens from subjects with pulmonary arterial hypertension and failed donor controls. 2) To provide the infrastructure to support human tissue based research in PAH via integrated cores devoted to Administration, Tissue Handling and Pathology, Cell Line Development, and Genomic Characterization and Cataloging. 3) To develop and implement an interface for consultation of research design and analysis to enhance broader scientific investigation.

描述（由申请人提供）：肺动脉高压（PAH）是一组异质性疾病，死亡率高、诊断困难、可用治疗有限且无法治愈。肺动脉高压突破计划 (PHBI) 致力于对患有多环芳烃 (PAH) 的人类肺部和对照进行全面的生物样本库，并以所采集样本的详细临床注释为支持。自 2006 年开始，PHBI 成功开发了一种新颖而独特的基础设施，其成功依赖于高度集成的大学站点网络的积极参与，这些站点在每个能力领域都拥有广泛的专业知识：PAH 临床护理的卓越性（包括患者应计费用）、肺移植、病理学、遗传学、基因组学和细胞分离。该应用程序的目标是将这些无与伦比的资源和独特的专业知识纳入 R24 支持的计划中，指导银行积累特定疾病组的样本，这些样本将与病理、遗传和基因组亚表型高度整合，这些亚表型不仅涉及肺部和血液样本，但现在扩展到衰竭的肺动脉高压心脏。该提案将与 NHLBI 目前在该领域支持的举措高度协同。我们基础设施和治理的灵活性将使我们能够做出明确的响应并与任何未来的 NHLBI 计划相结合。我们建议，这一独特的基础设施将发挥紧迫作用，推动关键发现向 PAH 患者的转化，并帮助新发现在该领域产生长期持久的影响。将追求以下具体目标： 该提案的具体目标 1) 为患有肺动脉高压和失败的供体对照受试者的标本建立一个综合的生物样本库。 2) 通过致力于管理、组织处理和病理学、细胞系开发以及基因组表征和编目的集成核心，提供基础设施来支持基于人体组织的 PAH 研究。 3) 开发和实施研究设计和分析咨询界面，以加强更广泛的科学研究。