Investigators in the Genetics and Molecular Biology Branch (GMBB) use molecular genetics techniques to identify and characterize genes involved in the normal development and function of many tissues. They also seek to identify...
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Investigators in the Genetics and Molecular Biology Branch (GMBB) use molecular genetics techniques to identify and characterize genes involved in the normal development and function of many tissues. They also seek to identify disease-associated gene alterations and to understand how these genetic defects contribute to a range of human diseases, including anemia, cancer, and immune disorders. To accomplish their goal, they study the genetics of blood cells, cells of the immune system, and epithelial cells. They are particularly interested in the study of stem cells, which have the dual capabilities of self-renewal and differentiation into multiple mature cell lineages. GMBB investigators also use yeast cells as a model system to study genome replication and factors that affect genome stability and aging. By studying how genes normally act and what the consequences are when they are mutated, GMBB investigators are attempting to develop effective treatments for genetic diseases. One important research focus of the Branch is gene transfer therapy: the introduction of genes into cells to correct the effects of mutations in cellular DNA. Although developing effective gene replacement therapies for genetic mutations has been a long, difficult endeavor, GMBB investigators are using the data and tools gleaned from the Human Genome Project to develop more sophisticated ways of monitoring delivery methods for these corrective, lifesaving approaches. By testing new approaches and technologies in cell lines and animal models, they are making new discoveries not only about factors that will be critical to the success of human clinical trials involving therapeutic gene transfer but also about the function of genes in health and disease.
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