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Mario Capecchi, PhD
Gene Targeting into the 21st Century: Modeling Human Disease from Cancer to Neuropsychiatric Disorders
Nobel Prize Laureate in Physiology or Medicine, 2007
Distinguished Professor of Human Genetics and of Biology
Howard Hughes Medical Institute Investigator
University of Utah School of Medicine
Salt Lake City, Utah
2 Riverside Circle, Roanoke, VA 24016
Gene targeting allows designed modifications of any chosen gene in the mouse. Using this technology allows the generation of mouse models for human disease. The models, once verified to recapitulate the human disease, can be used to understand the disease at the molecular, cellular, and organismic level not possible in humans. Once an understanding is reached, the model can in turn be used as a platform for developing new therapeutic protocols for the management of the disease.
Nobel Prize Laureate Mario Capecchi will provide examples of gene targeting developed from his own laboratory involving childhood cancers and neuropsychiatric disorders.
About the Speaker:
Mario Capecchi is a Distinguished Professor of Human Genetics and Biology and a Howard Hughes Medical Institute (HHMI) investigator at the University of Utah School of Medicine. Dr. Capecchi received his doctorate in biophysics from Harvard University, studying with Nobel Laureate James Watson.
Dr. Capecchi received the Nobel Prize in Physiology or Medicine in 2007 for his pioneering work on genetic engineering in mice, including the development of gene knockout technology. He is also a recipient of the Kyoto Prize, the National Medal of Science, the Albert Lasker Award for Basic Medical Research, and the Wolf Prize.
The gene-targeting technology that Dr. Capecchi developed has allowed scientists to engineer mice with conditions such as cancer, heart disease, Alzheimer’s disease, cystic fibrosis, and high blood pressure, revolutionizing the study of human disease. Scientists have used Dr. Capecchi’s discoveries to manipulate the genetic material of mice with unprecedented precision, creating desired mutations in virtually any gene for the study of that gene’s role in normal development or function and disease processes, as well as the development of new therapeutic approaches and treatments of disease.
Dr. Capecchi currently studies the molecular genetics of mammalian development, including birth of neurons in the brain, organ formation, patterning of the vertebral column, and limb development. He also contributes to the modeling of human disease in the mouse, from cancer to neuropsychiatric disorders. His lab studies human sarcomas in the mouse as well as the molecular genetic causes underlying trichotillomania, a human neuropsychiatric disorder on the obsessive-compulsive spectrum.