Mario Capecchi, a professor of human genetics at the University of Utah School of Medicine and a Howard Hughes Medical Institute investigator, is best known for his pioneering work on developing gene targeting in mouse embryo-derived stem (ES) cells. This technique allows the investigator to create mice with any desired mutation. "The power of this technology," says Capecchi "is that the investigator chooses both which gene to mutate and how to mutate it. The investigator has almost complete freedom on how to manipulate DNA sequences in the mouse genome."
Capecchi believes that, in the future, gene targeting will be used not only to "knock out" genes, but also to place genes under switches so the researcher controls when and where a gene is active in a mouse. In addition, this will allow scientists to evaluate in detail the function of any gene in living mice. Gene targeting is having an impact on almost all aspects of mammalian biology, including studies in development, immunology, neurobiology, cancer, and human congenital diseases.
An immediate application of gene targeting to medicine is in generating animal models for human diseases such as cystic fibrosis, atherosclerosis hypertension, and cancer. These models provide a unique opportunity to undertake an in-depth analysis of the pathology of the disease, and more important, provide the means for developing new therapeutic protocols.
Capecchi a member of the National Academy of Sciences, is a recipient of the Bristol-Myers Squibb Award for Distinguished Achievement in Neuroscience Research and Achievements in Medical Science.
Mario Capecchi reads THE SCIENTIST to keep abreast of political and technological developments that affect science. He also reads it for entertainment. "We, as scientists, are a funny breed of animal," he says. 'THE SCIENTIST helps us keep a balanced perspective on our attributes as well as our follies."
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