Fetal Biology and Therapy Training Program

描述(由申请人提供)：本T32培训计划提案的目标是促进新一代内科和外科胎儿专家的发展，这些专家将受到良好的培训，以开展创新的、科学严谨的实验室和临床研究，旨在寻求新的和有效的胎儿治疗策略。目前缺乏专门的专科培训和研究人员的准备，以便将胎儿作为病人进行治疗，这突显了这种方案的必要性。与此同时，将胎儿视为患者的看法正在形成势头，声称适用于胎儿的成功干预措施的数量迅速增加。因此，迫切需要这一领域训练有素的内科科学家进行发展研究和疗效和结果分析。该计划将支持学员进行长达3年的临床或实验室研究培训，培训级别为博士后奖学金。培训教师包括来自儿童医院和宾夕法尼亚大学医学院的16名教师和来自康奈尔大学的1名教师，他们拥有出色的资历，并专注于针对胎儿治疗的实验室或临床研究。这些导师将指导与胎儿治疗相关的基础实验室研究和/或以患者为导向的研究项目的受训人员。研究机会将包括四个主要的基础研究领域：1)胎儿干细胞疗法；2)胎儿基因疗法；3)胎儿伤口愈合；4)胎儿解剖畸形和胎儿肺生长。面向患者的研究机会将来自CHOP胎儿诊断和治疗中心，包括来自NICHD赞助的两个现有的多中心随机临床试验的机会，这些临床试验检查CHOP的脊髓脊膜膨出和双胞胎输血综合征的胎儿治疗，以及参与设计和实施胎儿治疗的各个方面的新研究。该计划包括所有研究方面的培训，包括生物统计学和生物伦理学，以及在胎儿治疗、分子生物学和相关领域的伦理学方面的具体计划。该计划得到了一系列机构资源的大力支持，包括CHOP胎儿诊断和治疗中心、CHOP GCRC和宾夕法尼亚大学研究生院和IHGT。本申请要求在第一年提供2个实习生名额，在第二年提供3个实习生名额，此后每年提供4个实习生名额。

DESCRIPTION (provided by applicant): The goal of this T32 Training Program proposal is to facilitate the development of a new generation of medical and surgical fetal specialists that will be well trained to carry out innovative and scientifically rigorous laboratory and clinical research directed toward novel and effective strategies for fetal treatment. The need for such a program is highlighted by the present lack of specific subspecialty training and preparation of investigators for treatment of the fetus as a patient. At the same time, the perception of the fetus as a patient is gaining momentum, and the number of claimed successful interventions applicable to the fetus has increased rapidly. There is thus a compelling need for well trained physician scientists in this field for developmental research and analysis of efficacy and outcomes. The Program will support Trainees for up to 3 years of clinical or laboratory research training at the postdoctoral fellowship level. The Training faculty includes 16 Faculty Mentors from The Children's Hospital and the University of Pennsylvania School of Medicine and 1 from Cornell University, who have outstanding credentials and a focused interest in laboratory or clinical research directed toward fetal treatment. These mentors will supervise Trainees in basic laboratory research and/or patient-oriented research projects related to fetal treatment. Research opportunities will include four major areas of basic research 1) Stem cell therapy in the fetus; 2) Fetal gene therapy; 3) Fetal wound healing; and 4) Fetal anatomic malformations and fetal lung growth. Patient oriented research opportunities will be derived from the CHOP Center for Fetal Diagnosis and Treatment and will include opportunities derived from two existing NICHD sponsored multicenter, randomized, clinical trials examining fetal treatment of myelomeningocele and Twin-Twin Transfusion Syndrome at CHOP as well as participation in the design and implementation of new studies of various aspects of fetal therapy. The Program includes training in all aspects of research, including biostatistics and bioethics, with a specific program in ethics in the context of fetal therapy, molecular biology, and related. The Program is strongly supported by access to a superb range of institutional resources, including the CHOP Center for Fetal Diagnosis and Therapy, the CHOP GCRC and the U. Penn Graduate School and IHGT. This request is made for 2 Trainee slots in year 1, 3 Trainee slots in year 2, and 4 Trainee slots each year thereafter.