CF Research Leads to 2007 Nobel Prize in Medicine

October 8, 2007

The 2007 Nobel prize in medicine, announced today, has been awarded to three scientists for their work in creating mouse models of disease.  All three of the scientists—Dr. Oliver Smithies of University of North Carolina, Sir Martin Evans of Cardiff University, and Dr. Mario Capecchi—have performed CF research that contributed to their award. 

The initial steps in this work began when Sir Evans isolated embryonic stem cells, making it possible to manipulate their genes to produce a "designer mouse." In work supported in part by the CF Foundation, Dr. Smithies at University of North Carolina used the stem cells to create one of the first animal disease models by disrupting the mouse CF gene. 

The resulting "CF mouse" has been widely used throughout the cystic fibrosis research community, resulting in a significant impact in understanding how CF affects the various organs. Because the mouse model is so useful, development of other CF mouse strains has followed. Teaming up with researchers at the University of Iowa, Dr. Capecchi engineered a mouse to carry the mutation that is the most common in people with cystic fibrosis. 

The altered mice are used by Sir Evans and many other CF researchers to study the effects of potential treatments that are geared to repair specific mutations. These studies have led directly to active clinical trials today. Sir Evans has also utilized the cystic fibrosis mouse for extensive studies in gene therapy, becoming the first to repair the CF deficit in a whole animal.

The genetic manipulation techniques developed by these scientists for creation of the CF mouse model have since been used for many other disease systems, revolutionizing the study of disease processes and drug development.