Basic Science Studies on Gene Therapy of Blood Diseases

描述（由申请人提供）： 这是自1999年以来取消的NIH T32 HL-07910的修订竞争性更新。其目的是继续培训下一代科学家在临床相关的医学领域的基因转移，以有效调节正常细胞生长和基因治疗。我们要求4个前和5个博士后职位和1个少数民族学生的短期前博士插槽。博士前和博士后学员将分别接受3-4年和2-3年的培训。我们从医学院的8个部门（微生物学/免疫学，生物化学/分子生物学，药理学/毒理学，医学/分子遗传学，医学，儿科，普通外科和泌尿外科）召集了24名富有成效/互动的研究人员组成的优秀团队，他们使用不同的细胞类型对博士前和/或博士后进行了多学科方法培训。培训重点是造血干细胞（HSC）和血细胞，但也包括其他细胞类型的基因修饰研究，以加强合作/培训经验。PI研究HSC、造血和基因替代，发表超过640篇论文，是众多编辑委员会/NIH/其他审查/咨询委员会成员，是ASH的现任候选人，培训了66名前/后医生。Co-PI在基因治疗和病毒载体生产领域得到认可，在这些领域发表了100多篇参考论文，是ASGT的预选赛，培训了10多名学生/研究员。PI/Co-PI是长期资助的基因治疗PPG的一部分，沿着该项目的其他6名导师，该项目包括一个国家认可和资助的临床级基因载体生产设施。我们的导师有广泛的合作，相互合作出版，所有实验室都在5-10分钟的步行距离内，并致力于培养学生的基因转移/治疗。培训需要一对一的互动，正式的委员会会议，实验室会议，特别研讨会系列，教学课程，道德培训和科学会议演示。一个内部和外部咨询委员会将监测学生/导师的进展。我们过去的学生受雇于学术界，生物技术和制药公司。这项培训将加强基因转移/治疗的未来，以基本了解和治疗疾病。

DESCRIPTION (provided by applicant): This is a revised competitive renewal of NIH T32HL-07910 undid since 1999. The aim is to continue training the next generation of scientists in the clinically-relevant medical area of gene transfer for effective modulation of normal cell growth, and gene therapy. We request 4 pre- and 5 postdoctoral positions and 1 short term pre-doc slot for a minority student. Pre- and post-doctoral trainees will be trained 3-4 and 2-3 years, respectively. We assembled an outstanding group of 24 productive/interactive investigators from 8 departments of the medical school (Microbiology/Immunology, Biochemistry/Molecular Biology, Pharmacology/Toxicology, Medical/ Mol-cular Genetics, Medicine, Pediatrics, General Surgery and Urology) who have trained pre- and/or post-doctorals with multidisciplinary approaches using different cell types. Training emphasis is on hematopoietic stem (HSC) cells and blood cells, but includes studies with other cell types for gene modification to enhance collaboration/training experiences. The PI studies HSC, hematopoietic, and gene replacement, published >640 papers ,is on numerous editorial boards/NIH/other review/advisory committees, is cun'ent Pres-Elect of ASH, trained 66 pre/post docs. The Co-PI is recognized in the gene therapy and viral vector production area, has published >100 refereed papers in these areas, is Pres-Elect of ASGT, has trained >10 students/fellows. The PI/Co-PI are part of a long-term funded PPG on gene therapy along with 6 other preceptors on the program, and the program includes a nationally- recognized and funded clinical grade gene vector production facility. Our preceptors have extensive collaboration , co-publish with each other, have all labs within 5-10 minute walking distance and are dedicated to training students in gene transfer/therapy. Training entails one-on-one interactions, formal committee meetings, lab meetings, special seminar series, didactic courses, ethical training and scientific meeting presentations. An Internal and External Advisory Committee will monitor student/mentor progress. Our past students are employed in academia, and biotech and pharmaceutical companies. This training will enhance the future of gene transfer/therapy for basic understanding and treatment of disease.