A Mouse (Trap) For Disease

By Rosalba Sacca, Ph.D. and Regina McEnery

About a quarter century ago, a trio of scientists developed a game-changing technology that enabled researchers to genetically engineer mouse strains with a particular gene missing. Finally, researchers had a tool to observe—in a backhanded way—how mice behaved when a critical piece of their DNA was missing.

And the era of the knockout mouse was born.

Laboratories began generating thousands of these so-called knockout strains—and many of them are now used to study major killers like cancer, central nervous system diseases, metabolic disorders and heart disease. But as is often the case in science, the animals were made on different strains and evaluated under different conditions. Moreover, there weren’t central places to validate KO strains in a standardized manner.