Core--Training

基于基因的疾病治疗方法在理论上是简单的；然而，事实证明，将其简化为实践是一项非同寻常的复杂任务，需要许多不同个人的专业知识。在临床基因治疗中，我们提出了独特的个体化培训方案。培训计划的目标将是将严格和杰出的博士后研究员培养成独立的研究人员，他们将继续推进基因治疗领域的发展。我们将为两名PEGT资助的学员(斯坦福大学和宾夕法尼亚大学各一名)提供为期两年的资助教育培训。培训将向拥有医学博士、医学博士或博士学位的个人开放。被该计划录取的成功受训人员将至少花费25%的精力进行血友病基因治疗的临床试验之一。他们剩余的时间可以花在基础/临床前研究、临床试验、数据管理设计等方面。作为培训的一部分，每个机构都有独特的授课教育机会。每名潜在候选人将由内部咨询委员会成员进行评估，并将以电话会议协商一致方式作出供资决定。费城的项目将由High博士和Kazazian博士共同监督，而斯坦福大学的项目将由Kay博士共同监督。培训计划的一个持续的目的是与导师和其他教职员工建立密切的关系，他们可以在研究员寻求额外资金和作为独立调查员的职位时为他/她提供指导。

Gene-based approaches to the treatment of disease are theoretically straightforward; reduction to practice, however, has proven to be an extraordinary complex undertaking that requires the expertise of many different individuals. We propose a unique individualized training program in clinical gene therapy. The goal of the training of the training program will be to graduate rigorous and outstanding post doctoral fellows into independent investigators who will continue to advance the field of gene therapy. We will offer two years of funded educational training for two PEGT funded trainees (one each at Stanford and CHOP/Penn). The training will be open to individuals with an MD, MD/PhD or PhD. The successful trainees accepted into the program will spend at least 25% of their effort on one of the clinical trials directed at gene-based therapies for hemophilia. The remainder of their time can be spent on some combination of basic/preclinical research, clinical trials, data management design. Unique didactic educational opportunities are available at each institution as outlined as part of the training. Each potential candidate will be evaluated by the Internal Advisory Committee members and decision for funding will be made by conference call consensus. The program in Philadelphia will e overseen jointly by Drs. High and Kazazian while the program at Stanford University will be overseen jointly by Dr. Kay. An ongoing purpose of the training program is the establishment of a close relationship with mentors and other members of the faculty who can provide guidance to the fellow as he/she seeks additional funding and a position as an independent investigator.