Gene- and Cell-based therapies for LVAD patients

针对 LVAD 患者的基因和细胞疗法

• 批准号: 7114607
• 负责人: DAVID ANDREW BULL
• 金额: $ 36.18万
• 依托单位: UNIVERSITY OF UTAH
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-09-30 至 2011-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7114607
• 关键词: angiogenesis; assistive device /technology; auxiliary heart prosthesis; bone marrow transplantation; circulatory assist; clinical research; clinical trials; cooperative study; drug administration rate /duration; gene delivery system; gene therapy; heart failure; heart function; human subject; human therapy evaluation; hypertrophic myocardiopathy; longitudinal human study; musculoskeletal regeneration; myocardium disorder; regional medical planning; stem cells; therapy design /development; vascular endothelial growth factors

DESCRIPTION (provided by applicant): SINCE 1985, the cardiology and cardiothoracic departments of the three major tertiary medical centers (University of Utah Hospitals, LDS Hospital, and Salt Lake City Veterans Affair Medical Center) have integrated into a single multihospital, multidepartment program in heart failure/cardiac transplant that serves a broad geographical region of the Western United States. This program, the Utah Transplantation Affiliated Hospitals (UTAH Network), services patients in Utah, Montana, Wyoming, Idaho, Nevada, Northern Arizona and Western Colorado. The UTAH network was the leading enroller for the REMATCH trial, a pivotal trial that established the efficacy of left ventricular assist devices (LVAD) as a destination therapy. Thus, the UTAH network is a clinical network with an extensive heart failure patient base, deep roots in the regional practice community, a long tradition of collaboration and coordination, and a proven history of recruiting patients for national heart failure trials. The UTAH investigators propose to treat LVAD patients with gene- and cell-based therapies that will induce angiogenesis, cardiac regeneration and repair, and enhance cardiac function. The LVAD patient population offers: 1) the unique opportunity to intensively study the functional and histopathological effects of these experimental medicines; 2) margin of safety for clinical trials involving novel gene- and cell-based therapies; 3) rapidly growing numbers of patients available for enrollment. Specifically the UTAH network proposes to treat heart failure patients undergoing LVAD placement with: Project 1- an ischemia-inducible VEGF expression vector using non-viral polymer based gene therapy. Project 2- direct injections of specific stem cell populations. The use of gene- and cell-based therapies at the University of Utah is supported by: 1) General Clinical Research Center with a 40 year old history; 2) Pioneering program developing non-viral polymer gene delivery systems 3) GMP clinical stem cell laboratory approved to support phase I and II protocols 4) Advance imaging core with expertise in LVAD and heart failure patients 5) Clinical Research Skills Development Program. (End of Abstract)

描述（由申请人提供）：