Fetal Biology and Therapy Training Program

描述（由申请人提供）：这个T32培训计划提案的目标是促进新一代医学和外科胎儿专家的发展，他们将接受良好的培训，以开展创新和科学严谨的实验室和临床研究，指导新的和有效的胎儿治疗策略。目前缺乏专门的亚专科培训和作为病人治疗胎儿的研究人员的准备，突出了对这样一个项目的需求。与此同时，胎儿作为病人的观念正在获得动力，声称成功的干预措施适用于胎儿的数量迅速增加。因此，迫切需要在这一领域训练有素的医师科学家进行发展研究和疗效和结果分析。该项目将为受训者提供长达3年的博士后水平的临床或实验室研究培训。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名教师导师，以及来自康奈尔大学的1名教师导师，他们具有杰出的资质，并对胎儿治疗的实验室或临床研究感兴趣。这些导师将指导学员进行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。研究机会将包括四个主要的基础研究领域：1)胎儿干细胞治疗；2)胎儿基因治疗；3)胎儿伤口愈合；4)胎儿解剖畸形与胎儿肺发育。以患者为导向的研究机会将来自CHOP胎儿诊断和治疗中心，将包括来自两个现有NICHD赞助的多中心随机临床试验的机会，这些试验检查了CHOP的脊髓脊膜囊肿和双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。该计划包括研究的各个方面的培训，包括生物统计学和生物伦理学，在胎儿治疗、分子生物学和相关的背景下，有一个特定的伦理计划。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。这一要求是在第一年提供2个培训生名额，在第二年提供3个培训生名额，此后每年提供4个培训生名额。

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.