Center of Excellence in Gene Therapy(PROGRAM PROJECT)

描述（由申请人提供） 此计划项目资助申请（PPG）是一个修订版的 我们最初为响应RFA HL-00-008而提交的提案， 为圣伊丽莎白的基因治疗卓越计划（PEGT）提供资金 医学中心（SEMC）/塔夫茨大学医学院（TUSM） 克利夫兰诊所外科的合作，以及 生物统计中心，乔治华盛顿大学，洛克维尔，马里兰州。的 PPG由三个临床项目和两个临床前项目组成， 由四个核心支持。Jeffrey M.博士Isner，主要研究者（PI） 全面负责PPG，将担任以下项目的项目总监 （临床）项目1，“VEGF基因转移治疗糖尿病神经病变。“博士。 道格拉斯洛索尔多将指导（临床）项目2，“VEGF基因转移到 预防冠状动脉再狭窄。（临床）项目3，VEGF基因 促进晚期心力衰竭患者血管生成的转移，”将 由克利夫兰诊所外科主任帕特里克麦卡锡博士指导 考夫曼心力衰竭中心。Ken沃尔什博士将作为项目 （临床前）项目4“心力衰竭的分子研究”主任。" 博士Takayuki Asahara将指导（临床前）项目5，“内皮 祖细胞基因转移。" 将提高PPG的研究生产力和职能能力 四个核心。核心A，即质粒核心，将提供临床级质粒 DNA载体，并将由沃尔什博士监督。核心B，行政 由Losordo博士领导的核心将提供关键的行政援助。 Drs. Losordo和沃尔什还将指导核心C，即培训核心。核心D， 数据管理核心，由Kathryn Hirst博士指导。研究助理 乔治·华盛顿大学生物统计学中心教授将 协助数据管理和分析，并确保遵守 所有适当的监管机构。 本PPG中概述的研究和组织旨在提供高 高质量和可靠的基因临床和临床前研究 这一转让将对科学和技术的发展作出有意义的贡献， 社区和公众。

DESCRIPTION (provided by the applicant) This Program Project Grant Application (PPG) is a revised version of a Proposal which we initially submitted in response to RFA HL-00-008 to seek funding for a Program of Excellence in Gene Therapy (PEGT) at St. Elizabeth's Medical Center (SEMC)/Tufts University School of Medicine (TUSM) with collaboration from the Cleveland Clinic Department of Surgery, and the Biostatistics Center, George Washington University, Rockville, Maryland. The PPG consists of three Clinical Projects and two Pre-clinical Projects, supported by four Cores. Dr. Jeffrey M. Isner, Principal Investigator (PI) with overall responsibility for the PPG, will serve as Project Director for (clinical) Project 1, "VEGF Gene Transfer for Diabetic Neuropathy." Dr. Douglas Losordo will direct (clinical) Project 2, "VEGF Gene Transfer to Prevent Coronary Artery Restenosis." (Clinical) Project 3, "VEGF Gene Transfer to Promote Angiogenesis in Patients with Advance Heart Failure," will be directed by Dr. Patrick McCarthy, Surgical Director of the Cleveland Clinic Kaufman Center for Heart Failure. Ken Walsh, Ph.D. will serve as Project Director for (pre-clinical) Project 4, " Molecular Studies of Heart Failure." Dr. Takayuki Asahara will direct (pre-clinical) Project 5, "Endothelial Progenitor Cell Gene Transfer." The research productivity and functional capacity of the PPG will be enhanced by four Cores. Core A, the Plasmid Core, will provide clinical grade plasmid DNA vector, and will be supervised by Dr. Walsh. Core B, the Administrative Core, directed by Dr. Losordo, will supply critical administrative assistance. Drs. Losordo and Walsh will also direct Core C, the Training Core. Core D, the Data Management Core, directed by Kathryn Hirst, Ph.D. Research Associate Professor of the Biostatistics Center, George Washington University will assist with data management and analysis, and will also ensure compliance with all appropriate regulatory bodies. The studies and organization outlined in this PPG are intended to provide high quality and credible clinical as well as pre-clinical research studies of gene transfer that will represent meaningful contributions to both the scientific community and lay public.