Training program in hematologic and oncologic diseases

描述（由申请人提供）：本提案要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博士前和博士后培训计划。该培训计划的基本前提是认识到并需要解决缺乏在儿科血液病和肿瘤疾病治疗研究方面受过培训的个人的问题，这些人也对将科学发现带入临床有深刻的认识。因此，培训计划的目标是提供对儿科血液学和肿瘤学疾病基本机制的严格理解，并具有明确的翻译重点。该项目在很大程度上借鉴了整合、多学科、创新和建立的研究生项目、博士、博士后培训项目和临床奖学金培训项目，涉及21名NIH资助的基础科学家和内科科学家作为核心指导教师和5名辅助临床教师。本项目招收有潜力的青年博士和博士后（2名预科生和4名博士后）候选人，重点招收少数民族候选人，在核心指导教师和辅助临床教师的直接指导下，专注于血液学和肿瘤学研究领域之一。研究领域包括儿童血液病，如白血病、范可尼贫血、骨髓增殖性疾病、凝血障碍和镰状细胞病；免疫缺陷疾病，如x连锁淋巴细胞增殖性疾病、严重联合免疫缺陷、wiskottaldrich综合征和常见可变免疫缺陷；治疗范可尼贫血、储存性疾病和镰状细胞贫血的分子基因治疗策略；以及针对儿童癌症的靶向治疗，如白血病/淋巴瘤、神经母细胞瘤、肉瘤和肌肉骨骼肿瘤。入选的项目教师在各种血液相关的研究活动中有着共同的研究兴趣和合作的历史，但他们从不同的角度研究血液和肿瘤疾病和治疗。使用小鼠模型与患者指导的举措相结合，代表了培训计划的独特优势之一。该资助将包括一个行政核心，由首席研究员、联合研究员、一个执行委员会、一个录取委员会、一个咨询委员会和一个外部咨询委员会组成。培训将结合一系列必修课程，包括伦理教育课程、实验血液学、疾病的免疫学机制、癌症治疗的作用、转化试验开发和支持实验室教育活动，以及一些选修课程，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。定期的研究静修、数据俱乐部、期刊俱乐部和系列研讨会将进一步提高学员的知识基础和项目的凝聚力，以补充导师指导的研究活动。每半年对每位学员的学习进度进行回顾和评估。

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.