MOLECULAR PEDIATRICS AND CHILD HEALTH

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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.