Core--Training

以基因为基础的疾病治疗方法在理论上是直截了当的;然而，实践证明是一项非常复杂的工作，需要许多不同个人的专业知识。我们提出了一个独特的临床基因治疗个性化培训计划。培训计划的培训目标是将严谨和优秀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。我们将为两名PEGT资助的受训人员提供两年的资助教育培训（斯坦福大学和CHOP/Penn各一名）。培训将开放给具有MD，MD/PhD或PhD的个人。成功接受该计划的学员将花费至少25%的精力用于针对血友病基因疗法的临床试验。他们的剩余时间可以花在基础/临床前研究，临床试验，数据管理设计的一些组合。每个机构都有独特的教学教育机会，作为培训的一部分。每个潜在的候选人将由内部咨询委员会成员进行评估，并通过电话会议协商一致作出资金决定。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。培训计划的持续目的是与导师和其他教师建立密切的关系，他们可以为研究员提供指导，因为他/她寻求额外的资金和独立调查员的职位。

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.