Basic Science Studies on Gene Therapy of Blood Diseases

描述（由申请人提供）： 这是一个培训计划的竞争性续期申请，以支持6名博士后学员（医学博士）。和/或博士学位）和3名博士生（M.D./哲学博士、或博士学位）重点是细胞和分子生物学/基因治疗的免疫学。 博士后和博士前学生的平均培训期分别为3年和4年。 22名经验丰富的导师将在高度活跃和互动的研究实验室中提供广泛的培训，这些实验室致力于对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进行最先进的研究，以及为其中一些疾病开发新型基因治疗方法的前景。 所有的导师都住在新建或翻新的宽敞的实验室，在4个基础科学部门，3个临床部门，并在两个研究中心，有几个交叉任命，在四个相邻的建筑物。培训计划的主要优势包括基因转移/基因治疗研究的良好记录，同行评审的出版物和导师的校外资助，以及三个NIH指定的国家载体实验室之一在校园内的存在。 由于各种导师之间积极、持续的科学合作，存在着进行多学科研究的充分机会。 一个重要的重点是促进和促进下一代生物医学研究人员的发展，致力于学术医学的科学事业，成为独立的调查人员，反过来，他们本身也将是富有成效和竞争力的。 现已采取多管齐下的综合培训办法，包括与教员密切和直接接触，与工作人员、访问科学家经常进行非正式互动，举办正式和非正式课程和研讨会，举行实验室会议，参加国家和国际科学会议，并将进一步予以强调。 与实验室合作的决定将由受训者和导师双方同意，受训者的科学发展将由一个由具有多学科研究兴趣的导师组成的研究委员会促进。 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，以及与布卢明顿的印第安纳州大学、西拉斐特的普渡大学和印第安纳波利斯的礼来公司的活跃研究者的频繁接触，所有这些都非常接近，以及国内和国际上的其他研究者。

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.