Grantees Win Lasker Award for Mouse Models of Human Disease

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Two long-time NIGMS grantees, Dr. Mario Capecchi and Dr. Oliver Smithies, have received the 2001 Lasker Award for Basic Medical Research. The award is "for the development of a powerful technology for manipulating the mouse genome with exquisite precision, which allows the creation of animal models of human disease," according to the Albert and Mary Lasker Foundation.

Working independently, Capecchi and Smithies created a method that has become an indispensable tool for genetics research. The method enables scientists to create "transgenic" mice--mice that contain genes from other organisms. If the transferred genes are involved in human diseases, the resulting mice can serve as model organisms for the study of those disorders. Scientists can use transgenic mice to learn about a disease in a mammal that is very similar to people, develop possible treatments, and test them with no risk to human patients. Capecchi and Smithies have already created transgenic mice to help them study cystic fibrosis and high blood pressure, and other scientists have used the mice to investigate diseases such as cancer and atherosclerosis.

Capecchi and Smithies' technique can also generate "knockout" lab mice, which help scientists pinpoint the actions of specific genes. To make a knockout mouse, researchers transfer a defective or altered version of a gene they want to study into a lab mouse. The inserted gene "knocks out," or displaces, the normal gene, and scientists can examine the modified gene's effects on the mouse.

"The tools these investigators have developed are both elegant and powerful," said NIGMS director Dr. Marvin Cassman. "They epitomize the goal of the National Institute of General Medical Sciences--to stimulate progress in the treatment and cure of disease through an understanding of basic biological processes."

Capecchi is the Distinguished Professor of Biology and Human Genetics at the University of Utah, and Smithies is the Excellence Professor of Pathology and Laboratory Medicine at the University of North Carolina at Chapel Hill School of Medicine. Also sharing the award with Capecchi and Smithies was Dr. Martin Evans of Cardiff University in Wales.

NIGMS has supported Capecchi's work since 1969 and Smithies' work since 1973.

The Lasker Award is one of the most prestigious science prizes in the United States. According to the Lasker Foundation, more than half of those honored with the Lasker Award for Basic Medical Research since 1962 have later received the Nobel Prize.

The Lasker Awards are scheduled to be presented in New York City on September 21.

More information on Capecchi, Smithies, and Evans' research is available in the Lasker Award press release at