MOLECULAR PEDIATRICS AND CHILD HEALTH

山区儿童健康研究中心的总体目标是 西奈医学院是提供选定的年轻儿科 研究人员拥有知识和核心实验室设施， 发展以分子儿科学为主题的基础科学研究项目 这将有助于他们作为独立的职业发展， 医生/科学家在主要研究者的指导下 和项目主任，一组既定的调查人员从 儿科学、生物化学、细胞生物学和解剖学、人类 遗传学、医学、微生物学、分子生物学、神经生物学和 生理学和生物物理学将提供基础科学研究 年轻儿科研究者的机会， 儿科医学，并提供未来独立的模型系统 研究项目。这些研究机会包括：1）分子 溶酶体贮积病的遗传学，2）逆转录病毒介导的基因 镰状细胞病和β-地中海贫血的治疗，3）组织特异性 珠蛋白和血红素的基因调控，以及遗传性 卟啉症，4）子宫内干细胞移植和基因转移到 血小板无力症中造血干细胞，5）粘膜 人B细胞免疫调节和淋巴因子调节 6）对流感病毒进行遗传操作，以用作 疫苗和载体，9）转基因小鼠在人类遗传学中的应用，11） 过氧化物酶体组装和过氧化物酶体紊乱的分子研究，12） 开发新疫苗的分子方法，13）分子 胶原蛋白在生长发育中的遗传学，14）神经内分泌学 压力和繁殖作为发展研究的模型系统， 和15）在动物模型系统中开发基因治疗策略。 共享核心实验室将提供寡核苷酸合成， 测序和组织培养系统，并建立敲除 小鼠模型，以促进年轻研究人员的努力。高- 高质量的，新培训的儿科医生将被招募到这个项目中 从地方和国家人才库中选出， 以马克思年轻的儿科研究人员和建立 研究者将参加每周一次的期刊俱乐部， 研讨会和年度研讨会。这个卓越的中心将 提供知识环境和核心实验室设施， 促进年轻儿科研究者的发展，他们将成为 并将其分子专业知识应用于当前和未来 儿科医学的问题。

The overall objective of the Child Health Research Center at the Mount Sinai School of Medicine is to provide selected Young Pediatric Investigators with an intellectual and core laboratory facilities to develop basic science research programs with a molecular pediatrics theme that will facilitate their career development as independent physician/scientists. Under the direction of the Principal Investigator and the Program Director, a group of Established Investigators from the Departments of Pediatrics, Biochemistry, Cell Biology and Anatomy, Human Genetics, Medicine, Microbiology, Molecular Biology, Neurobiology, and Physiology and Biophysics will provide basic science research opportunities for the Young Pediatric Investigators which have relevance to Pediatric medicine and provide model systems for future independent research projects. Among these are research opportunities in 1) molecular genetics of lysosomal storage diseases, 2) retroviral-mediated gene therapy of sickle cell disease and beta-thalassemia, 3) tissue specific gene regulation of globin and heme, and studies of the inherited porphyrias, 4) in utero stem cell transplantation and gene transfer into hematopoietic stem cells in Glanzmann's thrombasthenia, 5) mucosal immunoregulation and lymphokine regulation of human B cell differentiation, 6) genetic manipulation of influenza viruses for use as vaccines and vectors, 9) use of transgenic mice in human genetics, 11) molecular studies of peroxisomal assembly and peroxisomal disorders, 12) molecular approaches to the development of new vaccines, 13) molecular genetics of collagen in growth and development, 14) the neuroendocrinology of stress and reproduction as a model system for the study of development, and 15) development of gene therapy strategies in animal model systems. Shared core laboratories will provide oligonucleotides synthesis, sequencing and tissue culture systems, and the establishment of knock-out mouse models to facilitate the efforts of young investigators. High- quality , newly trained pediatricians will be recruited for this program from local and national pools and will be selected by an Advisory Committee. The Young Pediatric Investigators and the Established Investigators will participate in weekly Journal Club, and in monthly seminar, and an annual Research Symposium. This center of excellence will provide the intellectual environment and core laboratory facilities to enhance the development of Young Pediatric Investigators who will become independent and apply their molecular expertise to current and future problems in pediatric medicine.