Pulmonary Hypertension Breakthrough Initiative

肺动脉高压突破计划

• 批准号: 9103243
• 负责人: Serpil C. Erzurum
• 金额: $ 251.89万
• 依托单位: INDIANA UNIV-PURDUE UNIV AT INDIANAPOLIS
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-10-30 至 2018-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9103243
• 关键词: 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; Genetic; Genomics; Goals; Health; Heart; Human; Incidence; Informatics; Inherited; Institutes; Investigation; Lung; Lung Transplantation; Lung diseases; 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; Universities; Woman; Work; base; biobank; blood pump; clinical care; effective therapy; flexibility; heart cell; high throughput analysis; human tissue; hypertension control; meetings; member; mortality; novel; novel diagnostics; 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，治疗有限，无法治愈。肺动脉高压突破计划（PHBI）致力于对PAH和对照组的人类肺部进行全面的生物库，并得到累积标本的详细临床注释的支持。从2006年开始，PHBI成功地开发了一个新颖独特的基础设施，其成功依赖于一个高度集成的大学网站网络的积极参与，该网络在每个能力领域都具有广泛的专业知识：卓越的PAH临床护理（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利用这些无与伦比的资源和独特的专业知识，将其纳入R24支持的计划，以指导银行积累疾病特异性组的标本，这些标本将与病理学，遗传学和基因组亚表型高度整合，不仅与肺和血液标本有关，而且现在扩展到失败的PAH心脏。这项建议将与目前由国家HLBI在实地支持的倡议高度协同。我们的基础设施和治理的灵活性将使我们能够果断地响应并与任何未来的NHLBI计划相集成。我们建议，这种独特的基础设施将发挥推动关键发现向PAH患者转化的紧迫作用，并有助于在该领域产生长期影响的新发现。将努力实现以下具体目标： 提案的具体目标 1)建立肺动脉高压受试者和失败供体对照标本的综合生物储存库。 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综合核心，提供支持PAH中基于人体组织的研究的基础设施。 3)开发和实施研究设计和分析咨询接口，以加强更广泛的科学调查。