Training Grant in Neurodevelopmental Disabilities

项目概述：我们在费城儿童医院（CHOP）和宾夕法尼亚大学（U of P）建立的跨学科神经发育障碍机构培训计划的目的是培养医学博士和博士前/博士后研究人员，专注于导致智力迟钝和发育障碍的遗传和获得性疾病的研究。27位导师将参加该计划的第二次竞争性更新；其中20个主要设在CHOP， 7个主要设在university of P.所有这些机构都积极参与生物医学研究生教育，并通过相互研究项目和资助紧密联系在一起。除了有指导的研究培训，我们的课程强调：1)医学博士和博士的临床实习要求，2)通过明尼苏达大学研究生课程的正式课程，3)负责任的研究行为培训，4)生物统计学培训，5)涵盖各种重要生存技能的研讨会，包括科学写作，公开演讲，拨款写作研讨会，实验室管理和职业发展技能。在我们项目的前9年，我们接受了19名学员；4人是医学博士，14人是博士，1人是医学博士。这些受训者中有11人是女性，其中4人是代表性不足的少数民族。在完成培训的14人中，8人担任教职，2人继续在其他学术机构接受培训，1人是制药公司的高级研究科学家，1人是科学管理人员，1人是私人执业。我们要求继续支持4名博士后/年，并希望扩大我们的计划，包括支持2名博士前研究员/年。我们的目标仍然是提供一个不同于CHOP或uof P的其他培训项目，专注于发育性残疾的获得性和遗传原因。该项目受益于一个优秀的环境，将大量资源用于培训，基础生物医学研究，以及真正的“从实验室到床边”的转化研究方法。项目相关性：据估计，发育性残疾影响着美国10%的家庭。迫切需要愿意并能够运用现代研究方法来阐明这些疾病的发病机制和病理生理的研究人员，以便发现新的和更有效的治疗干预措施。本课程努力满足这一需求。

DESCRIPTION (provided by applicant): Project Summary: The aim of our interdisciplinary Institutional Training Program in Neurodevelopmental Disabilities, based at The Children's Hospital of Philadelphia (CHOP) and The University of Pennsylvania (U of P), is to train MD and PhD pre-/post-doctoral fellows in research focused on genetic and acquired disorders that cause mental retardation and developmental disability. Twenty-seven mentors will participate in this second competitive renewal of this program; 20 are based primarily at CHOP, and 7 primarily at the U of P. All are heavily involved in biomedical graduate education and are closely interlinked by mutual research projects and grants. In addition to mentored research training, our curriculum emphasizes: 1) a clinical practicum requirement for both MDs and PhDs, 2) formal course work through the U of P Graduate Studies Program, 3) training in responsible conduct of research, 4) training in biostatistics and 5) workshops that cover a variety of important survival skills, including scientific writing, public presentations, grant writing workshops, laboratory management, and career advancement skills. During the first 9 years of our program, we accepted 19 Trainees; 4 were MDs, 14 were PhDs, and 1 was a MD/PhD. 11 of these trainees were female and 4 of these trainees were under-represented minorities. Of the 14 who have completed training, 8 are in faculty positions, 2 are continuing their training at other academic institutions, 1 is a senior research scientist at a pharmaceutical company, 1 is a scientific administrator, and 1 is in private practice. We request continued support for 4 postdoctoral fellows/year and would like to expand our program to include support for 2 pre-doctoral fellows/year. Our goal remains focused on providing a training program unlike any other here at the CHOP or the U of P that is focused on acquired and genetic causes of developmental disability. This program benefits from being in an outstanding environment that commits substantial resources to training, to basic biomedical research, and to a true 'bench to bedside' approach to translational research. Project Relevance: It has been estimated that developmental disabilities affect 10% of all families in the USA. Researchers are urgently needed who are willing and able to apply modern research methods to elucidating the pathogenesis and pathophysiology of these disorders, so that new and more effective therapeutic interventions can be identified. This program strives to fill this need.