Training program in hematologic and oncologic diseases

描述（由申请人提供）：本提案请求支持辛辛那提大学医学院辛辛那提儿童医院医学中心（CCHMC）的儿科血液学和肿瘤学疾病的博士前和博士后培训项目。该培训计划的基本前提是，认识到并需要解决缺乏接受过儿科血液学和肿瘤学疾病治疗研究培训的人员的问题，这些人员也非常欣赏将科学发现带入临床。因此，培训计划的目标是提供一个明确的翻译重点儿科血液和肿瘤疾病的基本机制的严格理解。该计划在很大程度上借鉴了综合，多学科，创新和建立研究生博士学位。博士后培训计划，和临床奖学金培训计划，从7个部门在CCHMC，并涉及21个NIH资助的基础科学家和医生，科学家作为核心指导教师和5辅助临床教师。该计划招收有前途的年轻博士和博士后（2名博士前和4名博士后）候选人，重点是少数民族候选人的招聘，专注于核心指导教师和辅助临床教师的直接指导下的血液学和肿瘤学研究领域之一。研究领域包括儿科血液病，如白血病、范可尼贫血、骨髓增生性疾病、凝血障碍和镰状细胞病;免疫缺陷疾病，如X连锁淋巴细胞增生性疾病、严重联合免疫缺陷、Wiskott-Aldrich综合征和常见可变免疫缺陷;治疗范可尼贫血、胆积病和镰状细胞贫血的分子基因治疗策略;以及针对儿童癌症如白血病/淋巴瘤、神经母细胞瘤、肉瘤和肌肉骨骼肿瘤的靶向治疗。选定的项目教师分享研究兴趣，并在各种血液相关研究活动的研究中有合作的历史，但他们从不同的角度探讨血液学和肿瘤学疾病和治疗。使用小鼠模型结合患者导向的举措代表了培训计划的独特优势之一。赠款将包括一个行政核心，由首席研究员，Co-Pi，执行委员会，录取委员会，咨询委员会和外部咨询委员会组成。培训将结合联合收割机一系列必修课程，包括伦理教育课程，实验血液学，疾病的免疫机制，癌症治疗的作用，和转化试验开发和支持实验室教育活动，以及一些选修课，包括生物统计学，临床试验，功能基因组学和药物开发研讨会。定期的研究务虚会、数据俱乐部、期刊俱乐部和系列研讨会将通过进一步增强学员的知识基础和计划的凝聚力来补充导师指导的研究活动。每半年检讨及评估每名受训人员的进度。

Description (provided by applicant): This proposal requests support for a predoctoral and postdoctoral Training Program in Pediatric Hematologic and Oncologic Diseases in the Cincinnati Children's Hospital Medical Center (CCHMC), University of Cincinnati College of Medicine. The underlying premise of this Training Program is that there is a recognition of and need to address the lack of individuals trained in therapeutic research on pediatric hematologic and oncologic diseases who also have a deep appreciation for taking scientific discovery into the clinic. Therefore, the goal of the Training Program is to provide a rigorous understanding of the basic mechanisms of pediatric hematologic and oncologic diseases with a clear translational focus. The program draws heavily upon integrated, m ultidisciplinary, innovative, and established graduate progr ams, Ph.D. postodoctoral training programs, and clinical fellowship training programs from 7 divisions within CCHMC, and involves 21 NIH funded basic scientists and physician-scientists as core mentoring faculty and 5 ancillary clinical faculty. This program enrolls promising young doctoral and postdoctoral (2 predoctoral and 4 postdoctoral) candidates, with a emphasis on minority candidate recruitment, to specialize on one of the hematology and oncology research areas under the direct mentorship of a core mentoring faculty and an ancillary clinical faculty. The areas of research include pediatric hematologic diseases such as leukemia, Fanconi anemia, myeloproliferative disorders, coagulation disorders, and sickle cell disease, immuno- deficiency disorders such as X-linked lymphoproliferative disorder, severe combined immunodeficiencies, Wiskott-Aldrich syndrome, and common variable immunodeficiency, molecular gene therapy strategies treating Fanconi anemia, storage diseases, and sickle cell anemia, and targeted therapies against childhood cancers such as leukemia/lymphoma, neuroblastoma, sarcomas, and musculoskeletal tumors. The selected program faculty share research interests and have a history of collaboration in studies of various blood- related research activities, yet they approach hematologic and oncologic diseases and therapeutics from different perspectives. The use of mouse models combined with patient-directed initiatives represents one of the unique strengths of the Training Program. The grant will include an administrative core consisting of the Principal Investigator, Co-Pi, an Executive Committee, an Admission Committee, an Advisory Committee, and an External Advisory Committee. Training will combine a series of required curriculum including an ethical education course, Experimental Hematology, Immunological Mechanisms of Diseases, Action of Cancer Therapeutics, and Translational Trial Development and Support Laboratory Educational Activities, as well as a number of electives including Biostatistics, Clinical Trials, Functional Genomics, and Seminar in Drug Development. Regular research retreats, data clubs, journal clubs, and seminar series will serve to complement the mentor-directed research activities by further enhancing the knowled ge base of the trainees and cohesiveness of the program. The progress of each trainee will be reviewed and evaluated semi- annually.