Core--Training

这项建议的具体目的是为临床科学家提供高级培训，使他们能够从事与心血管基因治疗相关的翻译研究的学术生涯。大多数医生在完成内科或外科培训后，都不具备实施基本临床试验所需的知识或技能，更不用说利用基因治疗载体了。因此，实现这些目标的培训计划必须提供与人类基因治疗有关的临床试验、数据分析、医学伦理和监管事务的指导，以及基本的分子生物学技能。我们设计了为期两年的心血管基因治疗培训，每年为两名临床医生科学家培训。我们汇集了一批不同的教职员工，其中包括临床研究人员、分子生物学家、基因治疗师、免疫学家、药理学家、生物工程师、流行病学家、统计学家和生物伦理学家。在第一年，临床医生科学家实习生将参加由NHLBI资助的匹兹堡大学建立的临床研究培训计划(CRTP)。CRTP旨在教授设计和进行高质量临床研究所需的技能。我们将利用CRTP的核心课程和综合研讨会系列作为培训计划的一部分，以避免不必要的资源重复，并允许立即实施。此外，在第一年，每个受训人员将被分配到正在进行的临床心血管基因治疗项目之一，以获得早期接触到人类基因治疗试验。在第二年，学员将通过PCRN和GCRC参与管理积极的临床试验。匹兹堡临床研究网络(PCRN)是一个临床研究组织，为行业赞助的研究提供现场管理服务。综合临床研究中心(GCRC)提供匹兹堡大学进行临床研究和基因治疗试验所需的基础设施和支持人员。第二年的培训还将包括三个基因治疗研究模块，这些模块具有基本的课程和实验室经验，重点是基因治疗问题。总体教育目标是培养医生进行与心血管疾病相关的临床基因治疗研究，并在为临床试验寻求研究支持方面具有竞争力。预计每位学员将在完成培训计划的一年内提交一份独立的临床试验方案。

The specific aim of this proposal is to provide advanced training to clinician scientists to pursue an academic career in translational research related to cardiovascular gene therapy. Most physicians at the completion of their medical or surgical training do not possess the knowledge or skills needed to implement a basic clinical trial, let alone one utilization gene therapy vectors. Thus, a training program to accomplish these goals must provide exposure to the conduct of a clinical trial, data analysis, medical ethics, and regulatory affairs pertaining to human gene therapy, as well as basic molecular biology skills. We have designed a two-year cardiovascular gene therapy training for two Clinician Scientist trainees per year. We have assembled a diverse group of faculty members that include clinical investigators, molecular biologists, gene therapists, immunologists, pharmacologists, bioengineers, epidemiologists, statisticians, and bioethicists. During the first year, the Clinician Scientist trainees will participate in the NHLBI-funded Clinical Research Training Program (CRTP) established at the University of Pittsburgh. The CRTP aims to teach the skills necessary to design and conduct high quality clinical research. We will utilize the core curriculum and integrated seminar series of the CRTP as part of the training plan to avid unnecessary duplication of resources and allow of immediate implementation. Also, during the first year, each trainee will be assigned to one of the ongoing clinical Cardiovascular Gene Therapy projects to get early exposure to a human gene therapy trial. During the second year, the trainees will participate in the management of active clinical trials through the PCRN and the GCRC. The Pittsburgh Clinical Research Network (PCRN) is a clinical research organization that provides site management services for the conduct of industry-sponsored research. The General Clinical Research Center (GCRC) provides the infrastructure and support staff required for the conduct of clinical research and gene therapy trials at the University of Pittsburgh. The second year of training will also include three Gene Therapy Research Modules with basic coursework and laboratory experience focusing on gene therapy issues. The overall educational goals are to train physicians to conduct clinical gene therapy research related to cardiovascular disease and become competitive in seeking research support for clinical trials. It is expected that each trainee will submit an independent clinical trial proposal within a year of completing the training program.