Prereq: consent of the instructor. This course is designed to provide instruction in all aspects of the translation of basic research observations into clinical applications, using a case study model. The first case study will focus on sickle cell anemia. Lectures will review the molecular genetics of the disease and explore animal models for study of sickle cell anemia and will include laboratory exercises as appropriate. The course will address treatment strategies for sickle cell disease, including pharmacologic manipulation of globin gene expression, structural biology and rational drug design, and gene therapy. Additional topics related to clinical trials development will be covered including ethical issues, regulatory affairs, statistical con-siderations in clinical research, and outcome analysis. Required of all molecular medicine graduate students. 4 cr, 2nd sem.