These discoveries open up a new frontier of possibility, destroying flat-world theory. Research is now possible that never was before. Advanced animal models are the wind that makes the sailboat move. They accelerate and navigate the research. The implications for human health are enormous, clearly worthy of pursuit. Knock-out and knock-in mice have moved researchers into uncharted waters of possibility and promise.
Pathways of many hereditary conditions -- including obesity, high blood pressure and Parkinson's disease -- can be modeled and studied in a laboratory. Smithies and Evans recognized the potential of this discovery, using knock-out and knock-in mice to study cystic fibrosis.
This disorder devastates multiple organ systems in one out of every 3,700 babies born in the United States, shortening their life expectancy to 33 years. Ten million Americans carry the CF gene without ever experiencing its symptoms. But those who live with the disease have serious breathing difficulty, overproduction of mucus, intestinal blockage, liver damage and, finally, premature death.
Gene therapy, though in the early stages of research, aims to knock out human disorder-causing genes, among other functions. Eventually, this could mean knocking out cystic fibrosis and other inherited disorders.
Without Evans, Smithies and Capecchi, essential health studies could not proceed. These three researchers have blown the doors off laboratory expectations. Their accomplishments have earned them the Nobel Prize.
Countless others in the research community are employing these and
|SOURCE Foundation for Biomedical Research|
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