Fetal Biology and Therapy Training Program

胎儿生物学和治疗培训计划

• 批准号: 7417513
• 负责人: Alan W. Flake
• 金额: $ 23.43万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2004
• 资助国家: 美国
• 起止时间: 2004-05-01 至 2010-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7417513
• 关键词: Biology; Training Programs; fetal

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.

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医学和外科胎儿专家的发展，这些专家将接受良好的培训，以开展创新和科学严谨的实验室和临床研究，旨在实现新颖有效的胎儿治疗策略。由于目前缺乏专门的专科培训和研究人员对胎儿作为患者进行治疗的准备，因此突出了对此类项目的需求。与此同时，将胎儿视为患者的观念正在获得动力，声称适用于胎儿的成功干预措施的数量迅速增加。因此，迫切需要训练有素的医生科学家在这一领域的发展研究和疗效和结果的分析。该计划将支持实习生在博士后奖学金水平上进行长达3年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名教师导师和来自康奈尔大学的1名教师导师，他们具有出色的证书，并对针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基础实验室研究和/或与胎儿治疗相关的以患者为导向的研究项目。研究机会将包括基础研究的四个主要领域：1）胎儿干细胞治疗; 2）胎儿基因治疗; 3）胎儿伤口愈合; 4）胎儿解剖畸形和胎儿肺生长。以患者为导向的研究机会将来自CHOP胎儿诊断和治疗中心，并将包括来自两个现有的NICHD赞助的多中心，随机，临床试验检查胎儿治疗脊髓脊膜膨出和双胞胎输血综合征在CHOP以及参与设计和实施新的研究胎儿治疗的各个方面的机会。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，伦理学的具体方案。该计划得到了广泛的机构资源的大力支持，包括CHOP胎儿诊断和治疗中心，CHOP GCRC和美国。宾州研究生院和IHGT。此请求是在第1年提出的2个培训生名额，第2年提出的3个培训生名额，以及此后每年提出的4个培训生名额。

项目成果

Early gestational gene transfer of IL-10 by systemic administration of lentiviral vector can prevent arthritis in a murine model.

通过全身施用慢病毒载体进行妊娠早期 IL-10 基因转移可以预防小鼠模型中的关节炎。

• DOI: 10.1038/gt.2011.23
• 发表时间: 2011
• 期刊: Gene therapy
• 影响因子: 5.1
• 作者: Roybal,JL;Endo,M;Radu,A;Zoltick,PW;Flake,AW
• 通讯作者: Flake,AW