Researchers Make Advances in Mouse Trials Science

A new discovery allows researchers to study human disease more efficiently and in more species.


Mouse photo via Shutterstock

Mouse models have long been a mainstay in scientific research, but their days may be numbered. A new discovery from the Whitehead Institute would make it easier for researchers to study disease not only in mice, but in other animal models.

The research, led by the Whitehead’s Rudolph Jaenisch, hinges on altering the way scientists cause gene mutations in mouse models. And since researchers rely on creating gene mutations that mirror human conditions to study diseases and treatments in animal models, this discovery could have a large impact on the research community. The new method goes something like this: Instead of inserting pieces of DNA into a mouse’s embryonic stem cells and then implanting the altered cells into a female mouse, as was previously the standard, Jaenisch and his team bypassed embryonic cells and used a system that mirrors the way the body’s natural bacteria attacks viruses. (Jaenisch will describe his technique, known as CRISPR, in more detail in the journal Cell next week.) He says in a report from the Whitehead:

“This new method is a game changer,” says Jaenisch, who is also a professor of biology at MIT. “We can now make a mouse with five mutations in just three to four weeks, whereas the conventional way would take three to four years. And it’s rather straightforward, probably even easier than the conventional way.”

The research also opens up another door: That to using animals other than mice as models. Mice and rats have been used so frequently in the past because they are on the short list of species that have the embryonic stem cells formerly required for successful gene mutation. But if those cells are no longer needed, the list grows longer. The report quotes Hui Yang, a researcher on the team:

“This breaks down the definition of model organism,” says Wang, a postdoctoral researcher in Jaenisch’s lab. “So now, even with limited resources, any animal with established embryo manipulation procedures could be the subject of genome engineering. With many of the animals’ genomes that have been sequenced, we could use this technology to establish efficient genetic manipulations in more species, to study the unique biology of each, and to learn more about evolution.”

While the possibility of expanding animal research further will likely be met with some ethical push back, it is encouraging to see how much thought is being put into mouse trials, one of the most heavily-used but often flawed methods in medicine.