Basic Science Studies on Gene Therapy of Blood Diseases

描述(由申请人提供)： 这是一个培训项目的竞争性续签申请，旨在支持6名博士后实习生(医学博士和/或博士)。博士后3人(医学博士/博士或博士)每年重点研究细胞和分子生物学/免疫学的基因治疗。博士后和博士后研究生的平均培训期分别为3年和4年。22位著名的教授将在高度活跃和互动的研究实验室提供广泛的培训，从事各种人类疾病的细胞、分子生物学和免疫学方面的基本方面的最新研究，以及为其中一些疾病开发新的基因治疗方法的前景。所有的导师都被安置在新建或翻新的宽敞的实验室里，这些实验室位于4个基础科学系、3个临床科室和4个毗邻建筑的两个研究中心，其中有几个交叉预约。该培训计划的主要优势包括在基因转移/基因治疗方面的良好研究记录、同行评议的出版物和教师的外部资金，以及校园内存在的三个由NIH指定的国家媒介实验室之一。由于不同研究人员之间积极、持续的科学合作，存在着大量从事多学科研究的机会。重点是促进和促进下一代生物医学研究人员的发展，他们致力于学术医学的科学事业，成为独立的研究人员，他们本身将具有生产力和竞争力。已经采取了多管齐下的综合培训办法，包括与教官密切和直接接触、与工作人员、来访科学家、正式和非正式课程和研讨会、实验室会议以及参加国家和国际科学会议进行频繁和非正式的互动。与实验室合作的决定将由受训者和受训者双方同意，受训者的科学发展将由一个由具有多学科研究兴趣的受训者组成的研究委员会促进。可用的主要资源包括国际公认的临床和基础科学研究项目、最先进的基础设施，以及与位于布鲁明顿的印第安纳大学、位于西拉斐特的普渡大学和位于印第安纳波利斯的礼来公司的现役调查人员的频繁接触，所有这些都是非常接近的，以及其他国内外的调查人员。

DESCRIPTION (provided by applicant): This is a competitive renewal application for a training program to support 6 postdoctoral trainees (M.D. and/or Ph.D.) and 3 predoctoral students (M.D./Ph.D., or Ph.D.) per year with a strong emphasis on cell and molecular biology/immunology of gene therapy. The average training period for postdoctoral and predoctoral students will be 3 and 4 years, respectively. Twenty two well-established preceptors will provide extensive training in highly active as well as interactive research laboratories pursuing state of the art research on fundamental aspects of cellular, molecular biological, and immunological aspects of a variety of human diseases, and the prospects of developing novel gene therapy approaches for some of these disorders. All of the preceptors are housed in newly built or renovated spacious laboratories in 4 basic science departments, 3 clinical departments, and in two research centers, with several cross-appointments, in four adjoining buildings. Major strengths of the training program include a strong track record of research on gene transfer/gene therapy, peer-reviewed publications and extramural funding of the preceptors, and the existence of one of three NIH-designated National Vector Laboratories on campus. Ample opportunities for pursuing multi-disciplinary research exist due to active, ongoing scientific collaborations among various preceptors. A significant emphasis is placed to promote and foster the development of the next generation of biomedical researchers committed to scientific careers in academic medicine to become independent investigators who, in turn, will themselves be productive and competitive. A multi-pronged approach for comprehensive training encompassing close and direct contact with preceptors, frequent, informal interactions with staff members, visiting scientists, formal and informal courses and seminars, laboratory meetings, and participation in national and international scientific meetings, is already in place and will be further emphasized. The decision to associate with a laboratory will be by mutual consent of the trainee and the preceptor, and the scientific development of the trainee will be facilitated by a research committee composed of preceptors with multi-disciplinary research interests. Major resources that are available include internationally recognized clinical and basic science research programs, state of the art infra-structure, and frequent contacts with active investigators at Indiana University in Bloomington, Purdue University in West Lafayette, and Eli Lilly and Company in Indianapolis, all in close proximity, and other investigators nationally as well as internationally.