Gene Editing Tool for Studying Single-Base Mutations in Cancer

Scientists from Weill Cornell Medicine genetically engineered mice to enable the study of single base mutation in the genetic code. Their GE mice can be used by other researchers to help speed up progress toward personalized cancer treatment.

A change in a single base or “letter” can greatly affect the condition of a cancer patient. However, instead of single base mutations, the majority of studies involve deleting an entire gene. To focus on specific alterations, the researchers from Weill Cornell Medicine developed a precise gene editing tool to assess the impact of specific genetic changes in nonclinical models.

The researchers' GE mice have an enzyme that allows them to alter a letter in the genetic code of mice. The enzyme can also be switched on or off by providing the mice with doxycycline antibiotics, which will reduce the unplanned genetic changes that will occur over time. The team can also develop miniature versions of lung, intestine, and pancreas tissue, known as organoids.

"One mouse model allows you to do two things: test the effects of a mutation in cancer initiation, progression, or treatment response in mice and take a closer look at the associated molecular or biochemical changes using organoids," said Dr. Lukas Dow, one of the authors of the study.

For more information, read at Nature Biotechnology.