Pioneering Gene Editing Approaches in Cystic Fibrosis Research
Cystic Fibrosis (CF) is a genetic condition that affects about 40,000 people in the U.S. and over 200,000 worldwide. It is caused by changes in a gene called CFTR, which provides the instructions for making a protein important for healthy lungs and digestion. While new medications called modulators help many people with CF, they don’t work for everyone—and they are not a cure.
At the Harrison Lab, we are exploring a different solution: gene editing. This approach aims to repair the CFTR gene itself so the body can make the protein it needs to work properly. In 2012, our lab was the first to show that gene editing could correct the most common CF-causing change in a lab model. Since then, we have been working on ways to fix many more of the hundreds of variants that cause CF.
Today, our research is focused on three big questions:
- Can we use gene editing to target most —if not all— of the different CF-causing variants?
- Do we need a unique approach for each variant, or can we find strategies where we can edit clusters of variants in the CFTR gene?
- How do we transition gene editing from preclinical models to clinical trials?
Our goal is to bring new hope to every person living with CF—especially those who don’t benefit from current treatments.
